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Redpin bags A round to advance Chantix-controlled gene therapies – FierceBiotech

Redpin Therapeutics has raised $15.5 million to bankroll chemogenetic R&D. The initial close of the series A positions Redpin to move a controllable gene therapy for the treatment of indications such as epilepsy and pain toward an IND.

New York-based Redpin is a gene therapy company with a difference. Like its peers, Redpin uses viral vectors to introduce genes into patients. Yet, while for traditional gene therapy companies that is the end of the process, for Redpin it is just the start. The genes inserted by Redpin encode for engineered ion channel proteins designed to bind to a particular drug, namely Pfizers Chantix.

When the patient takes Chantix, the molecule binds to the the ion channels now expressed by the neurons, either stimulating or silencing the cells based on what is needed to treat the disease they cause. As Redpin CEO Elma Hawkins notes, the approach differs from traditional drug development.

We sort of the do the opposite of what has been done in the pharmaceutical industry for decades and decades. Instead of making small molecules for a biologic target or receptor, we pick a drug and then we make a very, very specific receptor for it. As long as these two molecules, the receptor and the small molecule, engage, you will have an effect on the neuron, Hawkins said.

The approach could enable very direct control over cellular activities. And, as the inserted gene only has a therapeutic effect in the presence of Chantix, the approach could allow physicians to effectively turn the gene therapy on and off.

That potential has caught the attention of investors. 4BIO Capital, an advanced therapy specialist that raised a $50 million fund last year, led the round with Arkin Bio Ventures. The VC wing of Takeda also chipped in cash as did seed round investors New York Ventures and Alexandria Venture Investments.

Having spent the past year amassing preclinical data, Redpin will use the money to push toward an IND, although at this time the biotech is yet to commit to a timeline for getting into the clinic. Redpin has been more forthcoming about the broad focus of its lead program, which is designed for use in conditions such as pain and epilepsy that are characterized by hyperexcited neurons.

If you have a therapy that can 'calm that down,' that would be a terrific solution as youre acting at the site of the problem, Hawkins said. Redpin could equally use the same basic approach to activate neurons, opening up another set of indications that are potentially amenable to treatment using its Chantix-controlled gene therapies.

Whatever the goal, the basic idea is to design and introduce receptors that bind to Chantix. The oral drug, which Pfizer sells to help smokers quit, became integral to Redpins approach in part because it penetrates the brain effectively and is well tolerated at the low doses needed for the gene therapy application.

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Redpin bags A round to advance Chantix-controlled gene therapies - FierceBiotech

Gene Therapy for Frontotemporal Dementia With a GRN Mutation Gets Fast Track Status – Monthly Prescribing Reference

Home News Drugs in the Pipeline

The Food and Drug Administration (FDA) has granted Fast Track designation to PR006 (Prevail Therapeutics), an experimental gene therapy, to slow the progression of frontotemporal dementia with a GRN mutation (FTD-GRN).

FTD-GRN is a progressive neurodegenerative disease caused by mutations in the GRN gene. Patients with a GRN mutation have reduced levels of progranulin, a protein critical for lysosomal function, neuronal survival, and normal microglial activities. PR006 delivers a healthy GRN gene using an AAV9 vector to increase progranulin levels in these patients.

The Food and Drug Administration (FDA) recently accepted the Investigational New Drug application for PR006, allowing the Company to proceed with a phase 1/2 trial.

FTD-GRN progresses rapidly and there are currently no therapeutic options available, said Asa Abeliovich, MD, PhD, Founder and CEO of Prevail. We believe PR006 has the potential to fill this unmet medical need and make a significant impact for patients.

The FDA previously granted Orphan Drug designation to PR006 for the treatment of patients with frontotemporal dementia.

For more information visit prevailtherapeutics.com.

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Gene Therapy for Frontotemporal Dementia With a GRN Mutation Gets Fast Track Status - Monthly Prescribing Reference

Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies – BioSpace

TAMPA, Fla. & DUBLIN--(BUSINESS WIRE)-- Vycellix, Inc., an immuno-discovery cell & gene therapy company, and Avectas Limited, a cell engineering technology business, today jointly announced that the companies have entered into a collaboration agreement to develop proprietary approaches for cell-based immunotherapeutic products.

The companies will collaborate on the delivery of Vycellix's novel RNA immunomodulator VY-M using Avectas' cell engineering platform, Solupore. The collaboration will address current limitations for cell-based therapies, in particular with respect to the need to accelerate the manufacturing process, reduce the cost of manufacture, and ultimately improve patient outcomes.

"We are delighted to partner with Vycellix and join forces in the development of novel cell-based products," stated Michael Maguire, Ph.D., CEO of Avectas. "We believe Solupore will play a critical role in the manufacture of cell-based therapies and will support a path towards effective patient outcomes."

According to Vycellixs President, Douglas Calder, Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our product candidate, VY-M, to T cells and natural killer (NK) cells. We expect to accelerate the expansion-time of T cells and NK cells by decreasing the non-dividing lag time, resulting in much shorter vein-to-vein delivery-time to patients. The studies will be conducted at Avectas Dublin-based facility and at Karolinska Institutet, Stockholm, Sweden.

Both Vycellix and Avectas are collaborative partners within NextGenNK, a newly established competence center for development of next-generation NK cell-based cancer immunotherapies based at Karolinska Institutet, Stockholm, Sweden. It is envisioned that Vycellix and Avectas will further expand their collaboration within the NextGenNK constellation.

We are excited to see the NextGenNK Competence Center catalyzing interactions among its industrial partners to advance NK cell-based immunotherapies, said Hans-Gustaf Ljunggren, M.D., Ph.D., Director of the NextGenNK Competence Center. The present collaboration may pave the way for similar collaborations among NextGenNK partners.

About Vycellix, Inc.: Vycellix is a private, immuno-discovery, life science company at the forefront of innovation in the development of cell & gene-based therapies targeting indications in, but not limited to, hematology/oncology, autoimmunity/chronic inflammatory diseases, and organ/tissue transplantation.

The Companys portfolio of transformational platform technologies encompass novel tools urgently sought after to enable broad global adoption of advanced therapies including: 1) the ability to generate Universal Cells (VY-UC), without the need to alter expression of any of the cellular components that control self-recognition (HLA Class I or II), obviating the need for immune-suppressive drugs and redefining the path towards off-the-shelf therapies; 2) the ability to amplify cell-potency through the upregulation of internal cytotoxic mechanisms (VY-X); 3) the ability to accelerate the expansion of cells for immunotherapy by near-elimination of non-dividing lag time to leap forward to shorter vein-to-vein time with expanded cells (VY-M); and, 4) the ability to markedly enhance gene transduction levels using viral vectors with implications for autologous and allogeneic CAR-T and CAR-NK cell development (VY-OZ).

The Companys platforms were all discovered by scientists at the world-renowned Karolinska Institutet (KI) in Stockholm, Sweden. KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine. For more information, please visit the Companys website at: http://www.Vycellix.com and follow its Twitter feed at: @Vycellix.

About Avectas Limited: Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex-vivo manufacture of our partners' gene-modified cell therapy products, which will retain high in-vivo functionality. Our vision is to be a leading non-viral cell engineering technology provider, integrated into manufacturing processes for multiple autologous and allogeneic therapies, commercialized through development and license agreements. For more information, please visit the Company's website at http://www.avectas.com.

Forward Looking Statements: This press release contains forward-looking statements. All statements other than statements of historical facts are forward-looking statements, including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as plan, expect, anticipate, may, might, will, should, project, believe, estimate, predict, potential, intend, or continue and other words or terms of similar meaning. These statements include, without limitation, statements related to the pre-clinical, regulatory, clinical and/or commercial development and all anticipated uses of VY-OZ, VY-X, VY-M and VY-UC, and the Companys plans for seeking out-licensing opportunities for these assets. These forward-looking statements are based on current plans, objectives, estimates, expectations and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with immuno-discovery product development, including risks associated with advancing products to human clinical trials and/or ultimately regulatory and commercial success which is subject to the uncertainty of regulatory approval, market adoption and other risks and uncertainties affecting Vycellix and its development programs. Other risks and uncertainties of which Vycellix is not currently aware may also affect Vycellixs forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. Vycellix undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200325005026/en/

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Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace

Avectas and Vycellix Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies – BioSpace

DUBLIN and TAMPA, Florida, March 24, 2020 /PRNewswire/ -- Avectas, a cell engineering technology business andVycellix, Inc. an immuno-discovery cell & gene therapy company, today announced that the companies have entered into a collaboration agreement to develop proprietary approaches for cell-based immunotherapeutic products.

The companies will collaborate on the delivery of Vycellix's novel RNA immunomodulator VY-M using Avectas' cell engineering platform, Solupore. The collaboration will address current limitations for cell-based therapies, in particular with respect to the need to accelerate the manufacturing process, reduce the cost of manufacture, and ultimately improve patient outcomes.

"We are delighted to partner with Vycellix and join forces in the development of novel cell-based products," says Michael Maguire, PhD, CEO of Avectas. "We believe Solupore will play a critical role in the manufacture of cell-based therapies and will support a path towards effective patient outcomes."

According to Vycellix's President, Douglas Calder, "Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our new product candidate, VY-M, to T cells and NK cells. We expect to accelerate the expansion-time of T cells and NK cells by decreasing the non-dividing lag time, resulting in much shorter "vein-to-vein" delivery-time to patients." The studies will be conducted at Avectas' Dublin-based facility and at Karolinska Institutet, Stockholm, Sweden.

Both Avectas and Vycellix are collaborative partners within NextGenNK, a newly established competence center for development of next-generation NK cell-based cancer immunotherapies based at Karolinska Institutet, Stockholm, Sweden. It is envisioned that Avectas and Vycellix will further expand their collaboration within the NextGenNK constellation.

"We are excited to see the NextGenNK competence center catalysing interactions among its industrial partners to advance NK cell-based immunotherapies," says Hans-Gustaf Ljunggren, MD PhD, Director of the NextGenNK competence center. "The present collaboration may pave the way for similar collaborations among NextGenNK partners."

In February 2020, Avectas announced that it had entered an agreement with the Centre for Commercialization of Regenerative Medicine (CCRM) based in Toronto, Canada to accelerate the translation of Avectas' non-viral cell engineering platform (Solupore) into the clinic.

About Avectas:Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex-vivo manufacture of our partners' gene-modified cell therapy products, which will retain high in-vivo functionality. Our vision is to be a leading non-viral cell engineering technology provider, integrated into manufacturing processes for multiple autologous and allogeneic therapies, commercialized through development and license agreements. For more information, please visit the Company's website at http://www.avectas.com

About Vycellix:Vycellix, Inc.is a private, immuno-discovery, life science company at the forefront of innovation in the development of cell & gene-based therapies targeting indications in, but not limited to, hematology/oncology, autoimmunity/chronic inflammatory diseases, and organ/tissue transplantation.

The Company's platforms were all initially discovered by scientists at the world-renowned Karolinska Institutet (KI) in Stockholm, Sweden. KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine. For more information, please visit the Company's website at http://www.vycellix.com

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SOURCE Avectas; Vycellix

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Avectas and Vycellix Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace

Global Viral Vector and Plasmid DNA Manufacturing Market to Surpass US$ 2,205.6 Million by 2027 – CMI – Yahoo Finance

SEATTLE, March 25, 2020 /PRNewswire/ -- According to Coherent Market Insights, the global viral vector and plasmid DNA manufacturing market is estimated to be valued at US$ 427.2 million in 2019, and is expected to exhibit a CAGR of 22.8% over the forecast period (2019-2027).

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Key Trends and Analysis of the Global Viral Vector and Plasmid DNA Manufacturing Market:

Key trends in the market include increasing incidences of cancer, rising number of product launches, and increasing collaboration and acquisition activities by key market players.

According to World Health Organization (WHO), in 2018, around 9.6 million cancer deaths occurred globally. Over the last decade, development of gene therapy for the treatment of the cancer has increased significantly. Gene therapy treatment for cancer include transfer of foreign genetic material in the targeted cancer cell in the host's body. Various types of viral vectors and plasmid DNA such as retrovirus and HGF plasmidare used in the development of gene therapy.

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Increasing product launches and approvals by regulatory authorities are expected to drive growth of the global viral vector and plasmid DNA manufacturing market over the forecast period. For instance, in December 2017, Spark Therapeutics received the U.S. FDA approval to launch the LUXTURNA in the U.S. market. It is the first FDA approved gene therapy for treatment for an inherited retinal disease (IRD) and the first adeno-associated virus (AAV) vector gene therapy approved in the U.S.

Furthermore, key players operating in the market are focused on adopting acquisition, agreement, and collaboration strategies, in order to expand their product offerings in markets. For instance, in December 2017, Merck KGaA entered into a commercial supply agreement with bluebird bio, Inc., a clinical-stage biopharmaceutical company. According to the agreement, Merck agreed to manufacture viral vectors for bluebird's gene therapy products targeting the rare genetic disorders.

Key Market Takeaways:

Key players operating in the global viral vector and plasmid DNA manufacturing market include

Lonza Group AG, FinVector Vision Therapies, Cobra Biologics and Pharmaceutical Services, Sigma-Aldrich Co. LLC, VGXI, Inc., VIROVEK, SIRION Biotech GmbH, FUJIFILM Diosynth Biotechnologies U.S.A., Inc., Sanofi, Cell and Gene Therapy Catapult, Brammer Bio, and MassBiologics.

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Market Segmentations:

Did not find what you were looking for? Here are some other topics:

DNA AND RNA SAMPLE PREPARATION MARKET

DNA and RNA samples are necessary for variety of applications in drug research and development and cancer studies. High quality DNA and RNA samples are important for a wide variety of research and clinical applications. Biological studies require purified and isolated nucleic acids as the first step and in all recombinant DNA techniques. The extraction of nucleic acids from biological material requires cell lysis, inactivation of cellular nucleases, and separation of the desired nucleic acid from cellular debris.

Read more @ https://www.coherentmarketinsights.com/market-insight/dna-and-rna-sample-preparation-market-3620

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LIFE SCIENCE PRODUCTS MARKET

Life science products include laboratory supplies & accessories, cell culture & fermentation processes, cell therapy technologies, chromatography products, bioprocess filtration, fixed and live cell research through imaging and analysis, sample collection products, recombinant proteins, cell lines, and antibodies. These products are used for drug discovery, tissue engineering, drug screening, forensic testing, and genetic analysis.

Read more @ https://www.coherentmarketinsights.com/market-insight/life-science-products-market-3652

GLYCOBIOLOGY MARKET

Glycobiology involves study of structural aspects, biosynthesis, and biology of polysaccharides and how they function in an organism. Study of glycobiology has variety of application in areas such as drug discovery and development, diagnostic applications, therapeutic application, and industrial applications.Complex structure of glycan's and difficulty in its study, high costs of spectrometry and high performance liquid chromatography are expected to hinder growth of the market.

Read more @ https://www.coherentmarketinsights.com/market-insight/glycobiology-market-3639

Contact Us:RajShahCoherent Market Insights1001 4th Ave.#3200Seattle, WA 98154Tel: +1-206-701-6702Email:sales@coherentmarketinsights.com

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Global Viral Vector and Plasmid DNA Manufacturing Market to Surpass US$ 2,205.6 Million by 2027 - CMI - Yahoo Finance

Genprex Strengthens Leadership Team with the Appointment of Two Accomplished Life Science Executives – Yahoo Finance

Seasoned industry professionals underpinned by newly strengthened balance sheet to drive key clinical programs

Genprex, Inc. ("Genprex" or the "Company") (Nasdaq: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced the appointment of Catherine M. Vaczy as Executive Vice President and Chief Strategy Officer and Michael T. Redman as Executive Vice President and Chief Operating Officer. These appointments follow on the heels of the Companys Fast Track Designation for its lead drug candidate, its newly licensed gene therapy for diabetes and capital raise of over $26 million that will support funding its clinical programs. Both executives will play pivotal roles as the Company advances toward the start of new clinical trials later this year and pursues partnerships for the development of its gene therapies.

"We are delighted to welcome Catherine and Michael to our executive leadership team. They join us at an exciting time as we advance our lead drug candidate into key clinical programs in non-small cell lung cancer and pursue partnership opportunities for our oncology platform and newly licensed gene therapy technology for diabetes," said Rodney Varner, Chief Executive Officer of Genprex. "Catherine and Michael each have significant biotechnology company experience that enhances our capabilities and will be instrumental in advancing our programs. Their combined expertise in business development, corporate strategy, manufacturing, clinical trial strategy and operations and regulatory affairs will be of great value as we build Genprex into a leading gene therapy company bringing new treatment options to patients in need. We are fortunate to have raised over $26 million just prior to the current turmoil in the markets, significantly strengthening our balance sheet and well positioning us financially as we execute on our strategic plan."

Ms. Vaczy has more than 20 years of experience as a founder and senior executive of life science companies, serving as a strategic partner and business and legal advisor to senior leadership teams and boards. Most recently, Ms. Vaczy has provided strategic advisory services to early stage biotechnology companies. In 2005, Ms. Vaczy co-founded and served for ten years on the senior leadership team of NeoStem, Inc. (now Caladrius Biosciences), a Nasdaq-listed clinical stage biotechnology company that combined a leading cell and gene therapy process development and manufacturing organization (sold to Hitachi Chemical) with a development pipeline of cell therapy products. Prior to that, she was an early employee and served on the senior leadership team of Nasdaq-listed ImClone Systems Incorporated (sold to Eli Lily and Company), a pioneer in targeted cancer therapy, where she was instrumental in forging important strategic alliances, including a transformative $1 billion co-development deal for the Companys blockbuster drug, Erbitux. Earlier in her career, Ms. Vaczy was a practicing attorney in a nationally recognized law firm representing early stage life science and other technology companies. Ms. Vaczy received a BA degree from Boston College and a JD degree from St. Johns University School of Law.

Mr. Redman brings more than 30 years of experience in the life sciences industry to Genprex. He has held a variety of key executive roles at clinical-stage companies, where he focused on strategic business development and U.S. and worldwide manufacturing and clinical operations. He has been instrumental in the consummation of multiple strategic transactions in the biotechnology and pharmaceutical industries. From 2007-2019, Mr. Redman served as President, Chief Executive Officer and Director of Oncolix, Inc., a publicly traded clinical-stage biopharmaceutical company focused on developing therapies for womens and childrens cancers. During his tenure at Oncolix, he advanced the companys lead drug into human clinical trials, completed the in-licensing of a promising radiopharmaceutical drug for the treatment of bone-related cancers, and took the company public. Prior to that, he was the CEO of Bone Medical, Inc., an Australian-based clinical stage company developing oral peptide products for the treatment of osteoporosis. In 2001, he co-founded Opexa Pharmaceuticals, a company developing immunotherapies for a variety of diseases, and served as its President and CEO until 2005. Mr. Redman also held key management positions with Zonagen (now Repros Therapeutics, which is a part of Allergan), Aronex Pharmaceuticals, Biovail Corporation and American Home Products (acquired by Pfizer). Mr. Redman earned a BA in Biology from the University of Missouri and an MBA from the University of Phoenix.

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About Genprex, Inc.Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with serious diseases who currently have limited treatment options. Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches for patients with cancer and other serious diseases. The Companys lead product candidate, Oncoprex, is being evaluated as a treatment for non-small cell lung cancer (NSCLC). Oncoprex has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for Oncoprex immunogene therapy for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso). For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking StatementsStatements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies , on cancer and diabetes, regarding potential, current and planned clinical trials, regarding our possible strategic partnerships and regarding our financial resources. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer and diabetes; the timing and success of our clinical trials and planned clinical trials of Oncoprex, alone and in combination with targeted therapies and/or immunotherapies, and whether other potential product candidates, including our gene therapy in diabetes advance into clinical trials; our ability to enter into strategic partnerships and the success of those partnerships; the timing and success of obtaining FDA approval of Oncoprex and other potential product candidates; and the extent and duration of the current and future economic challenges we may face. These and other risks and uncertainties are described more fully under the caption "Risk Factors" and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200323005076/en/

Contacts

Genprex, Inc. (877) 774-GNPX (4679)

Investor Relations GNPX Investor Relations(877) 774-GNPX (4679) ext. #2investors@genprex.com

Media Contact Genprex Media RelationsKalyn Dabbs(877) 774-GNPX (4679) ext. #3media@genprex.com

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Genprex Strengthens Leadership Team with the Appointment of Two Accomplished Life Science Executives - Yahoo Finance

Codexis and Takeda partner on gene therapies for rare diseases – Pharmaceutical Technology

]]> The companies will work together on three initial programmes for gene therapies to treat rare disorders. Credit: PublicDomainPictures from Pixabay.

Takeda Pharmaceutical has signed a strategic collaboration and licence agreement with biotherapeutics developer Codexis to research and create gene therapies for rare disorders.

Codexis will use its CodeEvolver protein engineering platform to construct gene sequences encoding protein variants that could boost efficacy by improving activity, stability and cellular uptake.

Takeda will combine these transgenes and its gene therapy capabilities to develop candidates for treating various rare genetic diseases, including lysosomal storage disorders and blood factor deficiencies.

Codexis president and CEO John Nicols said: Our CodeEvolver platform technology enables the rapid engineering of novel genetic sequences that encode more efficacious proteins. The prospects of these improved sequences for the development of differentiated gene therapies for patients with rare diseases, therefore, holds great promise.

Takedas expertise in developing novel treatments for patients with rare genetic disorders, and its commitment to developing the best possible gene therapies, makes them an ideal partner for our growing Novel Biotherapeutics business unit.

The companies will work together on three initial programmes. Codexis will generate enzyme sequences that could be progressed as gene therapies into pre-clinical development.

Takeda will carry out the pre-clinical and clinical development, as well as commercialisation.

Apart from the three programmes, Takeda could launch up to four programmes for different target indications.

Codexis will obtain an upfront payment, research and development (R&D) fee reimbursement.

The company is also eligible for development and commercial milestone payments, and sales royalties on any commercial product resulting from the partnership.

Earlier this month, Takeda Pharmaceutical announced plans to develop a drug to treat Covid-19. The company will develop a polyclonal hyperimmune globulin (H-IG), referred to as TAK-888.

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Codexis and Takeda partner on gene therapies for rare diseases - Pharmaceutical Technology

Study reveals gene therapy may help in treating cardiac disease – The Siasat Daily

California: In a breakthrough study, researchers have found a potential treatment for life-threatening cardiac diseases by using gene therapy.

Danon disease is a very rare, life-threatening condition where the fundamental biological process of removing and recycling proteins does not work.

This impairment results in dysfunction of the heart, skeletal muscle, neurologic system, eyes, and liver. Most patients die or require heart transplants by the third decade of life.

In the study, which was published in Science Translational Medicine, researchers have identified a novel way to treat Danon disease using gene therapy.

Heart transplant is not always available for patients and does not treat the other organs affected in Danon disease. We knew we needed to find therapies specifically designed to address the underlying cause, said the lead researcher Eric Adler.

Danon disease is a result of mutations in the gene LAMP2. For nearly a decade, Adler and a team of researchers at UC San Diego Health have been working to determine whether gene therapy could provide a new treatment approach.

Gene therapy involves either replacing or repairing a gene that causes a medical problem or adding genes to help the body treat disease. In this case, Adler and the team focused on adding a specially designed gene that restores the LAMP2 function, resulting in improved cardiac and liver function.

We utilised mice that were a model for Danon disease and missing this specific LAMP gene. We applied gene therapy to a group of these mice and compared to mice that did not receive treatment, said Adler.

The mice that received gene therapy expressed positive results in heart, liver and muscle function. The hearts overall function of ejecting blood and relaxing improved, as did the bodys ability to degrade proteins and metabolism.

Danon disease is more common in males, and symptoms begin in early childhood or adolescence.

In many cases, the condition is inherited by a parent, typically the mother. We believe Danon disease is actually more common than we think, but it is often misdiagnosed, said Adler.

By utilising gene therapy, we were able to identify a possible new treatment approach other than a heart transplant. This study is a significant step for patients with Danon disease, Adler added.

Prior studies in Adlers lab have focused on using a patients skin cells to create stem cells. These stem cells were used to create a heart model, allowing researchers to study Danon disease at the cellular level.

The approach has provided new insight into the diseases pathology and led to the idea of using gene therapy. Our work is also proof that using stem cells to model diseases has great potential for helping develop new medicines, said Adler.

The next step, said Adler, is testing in patients with Danon disease. A Phase I clinical trial for safety and efficacy has begun.

This is the first trial using gene therapy to treat a genetic cardiac disorder and three patients are currently being treated, which means were that much closer to finding a cure for this terrible disease, and may be able to use similar methods to treat other diseases, said Adler.

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Study reveals gene therapy may help in treating cardiac disease - The Siasat Daily

TCR therapy an attractive alternative to CAR T for immunotherapy – Drug Target Review

Chimeric antigen receptor (CAR) T-cell therapies have produced encouraging clinical outcomes, demonstrating their therapeutic potential in mitigating tumour development. However, another form of T-cell immunotherapy based on T-cell receptors (TCR) has also shown great potential in this field. Here, Nikki Withers speaks to Miguel Forte who elaborates on the process and explains why he is excited about seeing an idea translate into an industrial proposition.

STIMULATING the natural defences of a persons immune system to kill cancer cells, known as immunotherapy, has become a novel and exciting approach to treat cancer. For example, the role of T cells in cell-mediated immunity has inspired the development of several strategies to genetically modify T cells, such as chimeric antigen receptor (CAR) T-cell therapy, to target cancer cells. In recent years, CAR T-cell therapy has received much attention from researchers and the press alike, and the landmark approval and clinical successes of Novartis Kymriah (the first FDA-approved treatment to include a gene therapy step in the United States) and Gilead/Kite Pharmas Yescarta (the first CAR T-cell therapy for adults living with certain types of non-Hodgkin lymphoma) has prompted a surge of further research. However, this approach which involves isolating cells from a patient, bioengineering them to express CARs that identify and attach to tumour cells and injecting them back into the patient has several limitations, according to Miguel Forte, former CEO of Zelluna Immunotherapy and currently CEO of Bone Therapeutics.

Forte has been working on a T-cell immunotherapy approach that primarily focuses on the T-cell receptors (TCRs). Similar to CAR therapies, TCR therapies modify the patients T lymphocytes ex vivo before being injected back into the patients body. However, they differ in their mechanisms for recognising antigens. CAR T-cell therapy can be compared to a policeman, with a photograph of the criminal, being able to identify them on the street, explained Forte. It is an artificial way of guiding those cells to the cancer when the cancer cells are in suspension. The difficulty with CAR is that it cannot always penetrate and deliver an effect in solid tumours. TCR therapy, which utilises the natural mechanisms that T cells use to recognise the antigen and therefore the cancer, is better suited to penetrate the tumour ie, the policeman is able to go inside the building where a criminal is hiding.

It is obviously more costly at the beginning of the development when you are fine tuning your process, compared to when you progress to a larger scale as you approach the market

Of note, this approach targets the TCR- peptide/major histocompatibility complex (MHC) interaction, which enables eradication of tumour cells. Intracellular tumour-related antigens can be presented as peptides in the MHC on the cell surface, which interact with the TCR on antigen-specific T cells to stimulate an anti-tumour response. Imagine you, or the cells, are not just a soldier in an army but a captain that can bring other immune cells into the mix. TCRs and these cells, once they go in, have a direct kill activity and an immunostimulatory activity to other cells to have a more comprehensive effect of killing the tumour cells. Forte concluded that this approach is scientifically appealing and could bring value to a large array of solid tumours.

The benefits of TCR therapies are evident; however, as with all new approaches, it is not without its challenges. The first relates to the manufacturing of these therapies; the process requires extracting patient material, changing it and then returning it to the patient. Unlike drug discovery with small molecules where you have an inert, well-defined, chemically-established component, with biologics you go up a notch in terms of complexity, Forte explained, adding that while small molecules are unidimensional, biologics are three-dimensional and, thus, more complex and challenging to manufacture. You need to remember that your product, the cells, are a living being. It is something that replicates, changes and responds to its environment. This makes it a lot more challenging to characterise and define the right specifications of the product. The initial challenge is to put in place a consistent and reliable manufacturing process.

Generating the necessary pre-clinical data can also prove challenging; studies are easier to conduct in animal models when you are working with chemical entities rather than human cells, according to Forte. Finally, when the product does get to clinic, there are elements of manufacturing, supply and logistics that can prove challenging; however, companies are starting to provide solutions for this. Working in cell and gene therapy we need to apply what we have done with other products, explained Forte. You need to adapt to the complexity and diversity of the product you have in hand. Here, you have a live product. Something that responds. It is similar to having a child; you can modulate it, but you can never fully control the behaviour of something you are shaping.

Bringing a new drug to market, from drug discovery through clinical trials to approval, can be a costly process, especially when developing cell-based therapies. These are more expensive than developing chemistry or biologics, but when biologics started to be developed, they were also very expensive, explained Forte. We are now seeing a reduction of those costs as more companies are developing products and consequently more solutions are surfacing.

Forte was involved in developing his first cell therapy product about 10 years ago. At this time, it was difficult; a lot of solutions you had to build in house. Nowadays, you can import this from solutions already available so you can concentrate on the specificity; for instance, the viral vector for gene editing your cells or the cytokine concentration for the expansion of your cells. He added that as these therapies grow, so too does the competition, resulting in reduced costs. However, the price and return on investment must correlate with benefit. It is obviously more costly at the beginning of the development when you are fine tuning your process, compared to when you progress to a larger scale as you approach the market.

The well-publicised success story of Emily Whitehead a six-year-old leukaemia patient who was one of the first patients to receive CAR T-cell therapy is a prime example of the success of immunotherapy treatments. Even though these patients may need to continue medications, they can live a relatively normal life. The gene- edited cells remain in the individual and continue to control the cancer by restoring the immune systems capabilities, said Forte. He hopes that similar results will be seen with TCR therapies: Hopefully, a significant fraction of patients will have a clinical and biological response that will reduce the tumour bulk, give them a quality life and remain doing so by controlling the cancer for a significant amount of time.

Forte concluded that the possibilities for TCR- based immunotherapies are exciting and hopefully products will be developed that will deliver an immediate and sustained effect in cancer patients.

About the author

MIGUEL FORTE

Miguel is currently the CEO of Bone Therapeutics and visiting Professor at the Lisbon University in Portugal. He also serves as Chief Commercialization Officer and Chair of the Commercialization Committee of the International Society of Cellular Therapy (ISCT) and is Member of Board of Directors of ISCT and ARM. Miguel was CEO of Zelluna Immunotherapy until the end of 2019. Miguel holds a masters degree from the Faculty of Medicine of the University of Lisbon, Portugal, a PhD in Immunology from the University of Birmingham, UK, an accreditation as Specialist in Infectious Diseases and a certificate on Health Economics of Pharmaceuticals and Medical Technologies (HEP). He is Fellow of the Faculty of Pharmaceutical Medicine of the RCP in the UK.

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TCR therapy an attractive alternative to CAR T for immunotherapy - Drug Target Review

89bio Appoints Healthcare Industry Veteran Steven Altschuler, MD, as Chairman of the Board of Directors – BioSpace

SAN FRANCISCO, March 25, 2020 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardio-metabolic diseases, today announced the appointment of Steven Altschuler, M.D., as chairman of its board of directors. Dr. Altschuler currently serves as managing director of Healthcare Ventures at Ziff Capital Partners. He brings 20 years of experience growing healthcare organizations in business and leadership capacities.

We are very pleased to welcome Steven as chairman of 89bios board of directors, said Greg Grunberg, chair of the nominating and corporate governance committee of 89bios board of directors. By any measure, Steven has distinguished himself throughout his career as an innovator and dynamic leader in the provider and biotechnology industries. We are incredibly fortunate to have him join the team and confident he is going to be a significant contributor to 89bio.

In his role at Ziff Capital Partners, Dr. Altschuler co-leads the firms efforts toward investing in, and facilitating the start-up and development of, companies with potentially transformative technologies that emphasize cellular and molecular approaches to human disease. He previously co-founded Spark Therapeutics to develop and commercialize the preclinical and clinical gene therapy programs advanced at the Childrens Hospital of Philadelphia (CHOP) and other institutions. Dr. Altschuler served as Spark Therapeutics board chair from its founding through its acquisition in 2019, which included leading the company through a successful transition to a public company and the launch of the first gene therapy for an inherited disease approved by the U.S. Food and Drug Administration and European Medicines Agency.

I joined the board of directors because of my enthusiasm for 89bios potentially differentiated fibroblast growth factor 21 (FGF21), BIO89-100, and its commercial potential in non-alcoholic steatohepatitis (NASH) and severe hypertriglyceridemia (SHTG), said Dr. Altschuler. I look forward to working closely with the team and the rest of the board to advance our work in liver and cardio-metabolic diseases.

Trained as a pediatric gastroenterologist, Dr. Altschuler formerly held CEO positions at CHOP, which is one of the nations leading childrens hospitals, and the University of Miami Health System. He is a member of the board of directors of WW (formerly Weight Watchers International), Orchard Therapeutics, AsclepiX Therapeutics, ImVaX and Platelet Biogenesis. He is also an independent trustee of the Brigham and Womens Physician Organization at Mass General Brigham. Dr. Altschuler holds a B.A. in mathematics and an M.D. from Case Western Reserve University.

Steven is an accomplished business leader who has led and served on the boards of numerous biotechnology companies, said Rohan Palekar, CEO of 89bio. We look forward to drawing upon his strategic, operational and clinical expertise as we continue to grow the organization and work towards developing differentiated medicines for patients with liver and cardio-metabolic diseases.

About 89bio89bio is a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardio-metabolic diseases. The companys lead product candidate, BIO89-100, is being developed for the treatment of NASH. The company also intends to develop BIO89-100 for the treatment of SHTG. BIO89-100 is a specifically engineered glycoPEGylated analog of FGF21 that is currently in a proof of concept Phase 1b/2a clinical trial in patients with NASH or NAFLD and a high risk of NASH. 89bio is headquartered in San Francisco with operations in Herzliya, Israel. Visit 89bio.com for more information.

Forward-Looking Statements Certain statements in this press release may constitute "forward-looking statements" within the meaning of the federal securities laws, including, but not limited to, 89bios expectations regarding plans for its clinical programs and clinical studies. Words such as may, might, will, objective, intend, should, could, can, would, expect, believe, design, estimate, predict, potential, develop, plan or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward-looking statements. While 89bio believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in 89bios filings with the U.S. Securities and Exchange Commission (SEC)), many of which are beyond 89bios control and subject to change. Actual results could be materially different. 89bio expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law.

Investor Contact:Ryan MartinsChief Financial Officerinvestors@89bio.com

Media Contact:Lori RosenLDR Communications917-553-6808lori@ldrcommunications.com

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89bio Appoints Healthcare Industry Veteran Steven Altschuler, MD, as Chairman of the Board of Directors - BioSpace

Genprex Bolsters Board of Directors, Appoints Three Biotechnology and Healthcare Industry Leaders – Insurance News Net

Board additions will enhance Companys ability to execute on key corporate initiatives

AUSTIN, Texas--(BUSINESS WIRE)-- Genprex, Inc. (Genprex or the Company) (Nasdaq: GNPX), a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes, today announced the appointment of three new members to its Board of Directors. The new appointments are designed to strengthen the Company as it executes on its key clinical programs, continues to build its pipeline, and evaluates future license or corporate partnership agreements. The new Board members include Brent Longnecker, Chief Executive Officer of Longnecker & Associates, Jose A. Moreno Toscano, Chief Executive Officer of LFB USA Inc, and William R. (Will) Wilson, Jr., Chairman, President and Chief Executive Officer of Wilson Land & Cattle Co.

This strengthening of the Board of Directors closely follows the Company receiving Fast Track Designation from the Food and Drug Administration (FDA) for its lead drug candidate, raising more than $26 million from institutional investors over the last few months, and licensing a new gene therapy drug candidate for diabetes from the University of Pittsburgh. Also, just yesterday, the Company announced that it strengthened its management team by adding two new senior executives, Catherine Vaczy as Executive Vice President and Chief Strategy Officer, and Michael Redman as Executive Vice President and Chief Operating Officer.

We are honored to have Brent, Jose and Will join our Board in what we believe is a transformational time for our Company, said Rodney Varner, Chief Executive Officer of Genprex. Their combined experience in regulatory compliance, business development, clinical trial management, and deal structuring will be invaluable as we continue to our develop gene therapy drug candidates and pursue partnerships for our drug candidates. The addition of these outstanding directors, together with our new senior executive hires and a significantly strengthened balance sheet, puts us in an excellent position to execute our plans.

Brent Longnecker has more than 30 years of experience in corporate governance, executive compensation, and risk management consulting for public, private, and non-profit organizations. Mr. Longnecker built one of the countrys leading privately-held executive compensation and corporate governance consultancies, serving both domestic and international markets. Mr. Longnecker has deep expertise in healthcare, energy, real estate, manufacturing, and financial companies, regularly consulting with boards of directors, CEOs, key executives, and advisors in many major industries. He is a prolific author on the subjects of executive compensation and corporate governance.

Jose A. Moreno Toscano brings to the Company over 20 years of experience in the pharmaceutical and biotechnology industries, building, developing and transforming organizations. Mr. Moreno Toscano has a successful track record of identifying and capitalizing on opportunities to drive exponential revenue growth and market expansion, revitalizing underperforming operations and establishing foundations for successful start-up operations. His experience includes strategic planning, corporate restructuring, business development, M&A, investor relations, and general management.

William R. Wilson, Jr. has more than 40 years of experience as an attorney, with legal experience spanning health care regulation, biotechnology, clinical trial management, nursing home licensing and regulation, physician accreditation, securities, corporate governance, and contractual matters. He previously served as Judge of the 250th District Court of Travis County, Texas, where he presided over civil litigation, as well as Assistant District Attorney for Dallas County, Texas.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and other serious diseases. Genprexs technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and other serious diseases who currently have limited treatment options. Genprex works with world-class institutions and collaborators to in-license and develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches for patients with cancer and other serious diseases. The Companys lead product candidate, Oncoprex, is being evaluated as a treatment for non-small cell lung cancer (NSCLC). Oncoprex has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for Oncoprex immunogene therapy for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso). For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding our possible commercial partnerships and regarding our financial resources. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of Oncoprex, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including our gene therapy in diabetes, advance into clinical trials; our ability to enter into strategic partnerships and the success of those partnerships; the timing and success of obtaining FDA approval of Oncoprex and our other potential product candidates; and the extent and duration of the current and future economic challenges we may face. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200324005120/en/

Genprex, Inc. (877) 774-GNPX (4679) Investor Relations GNPX Investor Relations (877) 774-GNPX (4679) ext. #2 [emailprotected] Media Contact Genprex Media Relations Kalyn Dabbs(877) 774-GNPX (4679) ext. #3 [emailprotected]

Source: Genprex, Inc.

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Genprex Bolsters Board of Directors, Appoints Three Biotechnology and Healthcare Industry Leaders - Insurance News Net

Cancer Gene Therapy Market Boosting the Growth Worldwide: Cancer Gene Therapy Market Dynamics and Trends, Efficiencies Forecast 2025 – Daily Science

In this new business intelligence Cancer Gene Therapy market report, PMR serves a platter of market forecast, structure, potential, and socioeconomic impacts associated with the global Cancer Gene Therapy market. With Porters Five Forces and DROT analyses, the research study incorporates a comprehensive evaluation of the positive and negative factors, as well as the opportunities regarding the Cancer Gene Therapy market.

With having published myriads of Cancer Gene Therapy market reports, PMR imparts its stalwartness to clients existing all over the globe. Our dedicated team of experts deliver reports with accurate data extracted from trusted sources. We ride the wave of digitalization facilitate clients with the changing trends in various industries, regions and consumers. As customer satisfaction is our top priority, our analysts are available 24/7 to provide tailored business solutions to the clients.

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The Cancer Gene Therapy market report has been fragmented into important regions that showcase worthwhile growth to the vendors Region 1 (Country 1, Country 2), region 2 (Country 1, Country 2) and region 3 (Country 1, Country 2). Each geographic segment has been assessed based on supply-demand status, distribution, and pricing. Further, the study provides information about the local distributors with which the Cancer Gene Therapy market players could create collaborations in a bid to sustain production footprint.

Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

Key points covered in the report

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Readers can get the answers of the following questions while going through the Cancer Gene Therapy market report:

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PMR is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

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Cancer Gene Therapy Market Boosting the Growth Worldwide: Cancer Gene Therapy Market Dynamics and Trends, Efficiencies Forecast 2025 - Daily Science

Serial child rapist sentenced to up to 126 years in jail; new gene therapy from UPMC could cure diabetes: Tod – PennLive

Listen to the latest episode at this link, or on your favorite app including Alexa, Apple, Google, Spotify and Stitcher. Episodes are available every morning on PennLive. Subscribe/Follow and rate the podcast via your favorite app.

A serial child rapist is going behind bars for life after being sentenced to up to 126 years in jail. Meanwhile, a couple is being charged with arson and insurance fraud after allegedly setting fire to their newspaper business. In Pittsburgh, a UPMC researchers new gene therapy could cure diabetes. Also, with Easter on the horizon, Cadbury is on the hunt for a different kind of bunny -- and one Pennsylvania llama is throwing its hat in the race.

Those are the stories we are covering in the latest episode of Today in Pa, a daily weekday podcast from PennLive.com and hosted by Julia Hatmaker. Today in Pa is dedicated to sharing the most important and interesting stories in the state.

Todays episode refers to the following articles:

Special thanks to Apple Podcast listener wildbill95 for leaving Today in Pa. a review. Heres what they wrote:

Great start to the day! Julia provides the most-needed details on the days news, and makes a great way to begin the morning. Just as important, Julia gives us the lede to stories we now know to look for the in-depth article on the website.

Thanks, Julia, for giving us a boost on a ho-hum morning!"

If you enjoy Today in Pa, consider leaving us a review on Apple Podcasts or on Amazon. Reviews help others find the show and, besides, we like to know what you think of the program.

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Serial child rapist sentenced to up to 126 years in jail; new gene therapy from UPMC could cure diabetes: Tod - PennLive

Visiongain Report Looks at Opportunities Within the $23bn Cell Therapy Technologies Market – Yahoo Finance

Cell Therapy Technologies Market Forecast 2020-2030

LONDON, March 25, 2020 /PRNewswire/ -- Consumables, Equipment, System & Software, Cell Processing, Cell Processing Equipment, Single Use Equipment, Cell Preservation, Distribution, Handling, Process Monitoring & Quality Control, Human Cells, Animal Cells, Life Sciences & Research Companies, Research Institutes

Visiongain estimates that the global cell therapy technologies market will grow at a CAGR of 15% in the first half of the forecast period. In 2020, North America is estimated to hold 38% of the global cell therapy technologies market.

How this report will benefit you

Read on to discover how you can exploit the future business opportunities emerging in this sector.

In this brand new201-page reportyou will receive104 tables and 110 figures all unavailable elsewhere.

The 201-page Visiongain report provides clear detailed insight into the cell therapy technologies market. Discover the key drivers and challenges affecting the market.

By ordering and reading our brand-new report today you stay better informed and ready to act.

To request sample pages from this report please contact Sara Peerun at sara.peerun@visiongain.com or refer to our website: https://www.visiongain.com/report/cell-therapy-technologies-market-forecast-2020-2030/#download_sampe_div

Report Scope

Global Cell Therapy Technologies Marketfrom2020-2030

Forecast of the Global Cell Therapy Technologies Market byType of Product: Consumables Equipment:Cell Processing Equipment, Single Use Equipment, Other Equipment System & Software

Forecast of the Global Cell Therapy Technologies byProcess Type: Cell processing Cell preservation, distribution and handling Process monitoring and quality control

Forecast of the Global Cell Therapy Technologies byCell Type: Human cells:Stem cells, Differentiated cells Animal cells

Forecast of the Global Cell Therapy Technologies byEnd User: Life Sciences and Research Companies Research Institutes

This report provides individual revenue forecasts to 2030 for thesenational markets: The US Canada Mexico UK Germany France Italy Spain Japan China India South Korea Singapore Malaysia Russia Brazil Argentina UAE South Africa Nigeria Mexico

Our study discusses the selectedleading companiesthat are the major players in the respiratory inhalers market: GE Healthcare Lonza Group Merck KGaA Terumo Bct, Inc. Thermo Fisher Scientific, Inc. & Other Companies

For the leading companies, we feature product portfolios, business segment breakdowns, recent developments & key expansion strategies etc.

This report discussesfactors that drive and restrainthis market. As well asopportunitiesandchallengesfaced by this market.

This report discusses thePorter's Five Forces Analysisof the Cell Therapy Technologies Market.

Key questions answered by this report: How is the Cell Therapy Technologies Market evolving? What is driving and restraining factors of the Cell Therapy Technologies Market? What are the market shares of each segment of the overall Cell Therapy Technologies Market in 2020? How will each Cell Therapy Technologies submarket segment grow over the forecast period and how much revenue will these submarkets account for in 2030? How will the market shares for each Cell Therapy Technologies submarket develop from 2021 to 2030? What will be the main driver for the overall market from 2021 to 2030? Will leading national Cell Therapy Technologies Markets broadly follow the macroeconomic dynamics, or will individual national markets outperform others? How will the market shares of the national markets change by 2030 and which geographical region will lead the market in 2030? Who are the leading players and what are their prospects over the forecast period? How will the industry evolve during the period between 2020 and 2030?

Story continues

To request a report overview of this report please contact Sara Peerun at sara.peerun@visiongain.com or refer to our website: https://www.visiongain.com/report/cell-therapy-technologies-market-forecast-2020-2030/

Did you know that we also offer a report add-on service? Email sara.peerun@visiongain.comto discuss any customized research needs you may have.

Companies covered in the report include:

Affymetrix, Inc.AkouosAllCellsApplikon Biotechnology Inc.ATLATL CentreAutolus LimitedBeckman Coulter, Inc.Becton, Dickinson and CompanyBioengineering AGBiological IndustriesBioWa, IncBrammer BioC.R. Bard, Inc.CaridianBCT, IncCell and Gene Therapy Asia Technology CentreCentre for Process Innovation (CPI)CMC Biologics (Asahi Glass Co.)Cobra BiologicsCocoon PlatformCryoportDanaher CorporationDiNAQOR AGEMD Performance MaterialsEMD SeronoEppendorf AGEuropean Molecular Biology Laboratory (EMBL)Finesse Solutions, Inc.Flexsafe RM TXFloDesign SonicsFlowJo, LLCFood and Drug Administration (FDA)Gamida CellG-CON ManufacturingGE healthcareGenScriptInfors HTIntegrated DNA Technologies, Inc.LaVision BioTecLonza Group, GE HealthcareMassachusetts Eye and Ear (MEE)Meissner Filtration Products, Inc.Merck KGaAMerck SeronoMesoblastMilliporeSigmaMiltenyi BiotecNova BiomedicalPall Corporation (Pall)Patheon N.V.Penn State UniversityPharmaCell B.VSartorius AGSartorius Stedim BiotechScinogySelecta Biosciences, IncSiemensSolaris BiotechStafa Cellular TherapyStafaCTStemcell TechnologiesTerumo Bct, Inc. (A Subsidiary of Terumo Corporation)Thermo Fisher Scientific, Inc.Tillotts Pharma AGTranstem LabUniCAR TherapyWorld Courier

To see a report overview please e-mail Sara Peerun on sara.peerun@visiongain.com

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Global Stem Cell Technologies and Applications Market 2019-2029

Biobanking Market Forecasts 2019-2029

Biologics Market Trends and Forecasts 2019-2029

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Global Precision Medicine Market Forecast 2019-2029

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Visiongain Report Looks at Opportunities Within the $23bn Cell Therapy Technologies Market - Yahoo Finance

Gene therapy – Mayo Clinic

Overview

Gene therapy involves altering the genes inside your body's cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body's form and function, from making you grow taller to regulating your body systems. Genes that don't work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can't easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells' genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren't the only vectors that can be used to carry altered genes into your body's cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Dec. 29, 2017

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Gene therapy - Mayo Clinic

gene therapy | Description, Uses, Examples, & Safety …

Discover how gene therapy can treat diseases caused by genetic mutations such as cystic fibrosisGene therapy seeks to repair genetic mutations through the introduction of healthy, working genes.Encyclopdia Britannica, Inc.See all videos for this article

Gene therapy, also called gene transfer therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype.

Read More on This Topic

cancer: Gene therapy

Knowledge about the genetic defects that lead to cancer suggests that cancer can be treated by fixing those altered genes. One strategy

Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome. Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germline gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If that is achieved, those cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germline gene therapy has been achieved experimentally in animals but not in humans.

Scientists have also explored the possibility of combining gene therapy with stem cell therapy. In a preliminary test of that approach, scientists collected skin cells from a patient with alpha-1 antitrypsin deficiency (an inherited disorder associated with certain types of lung and liver disease), reprogrammed the cells into stem cells, corrected the causative gene mutation, and then stimulated the cells to mature into liver cells. The reprogrammed, genetically corrected cells functioned normally.

Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. Few clinical trials of gene therapy in humans have satisfied all those conditions, often because the delivery system fails to reach cells or the genes are not expressed by cells. Improved gene therapy systems are being developed by using nanotechnology. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed.

Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Some objections to gene therapy are based on the view that humans should not play God and interfere in the natural order. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Benefits, however, would also be passed on indefinitely. There also has been concern that the use of somatic gene therapy may affect germ cells.

Although the successful use of somatic gene therapy has been reported, clinical trials have revealed risks. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. In 2000 researchers in France announced that they had successfully used gene therapy to treat infants who suffered from X-linked SCID (XSCID; an inherited disorder that affects males). The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Those outcomes highlight the difficulties foreseen in the use of viral vectors in somatic gene therapy. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response.

Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. While some have argued that gene therapy is eugenic, others claim that it is a treatment that can be adopted to avoid disability. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples hopes for new treatments that may never materialize.

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gene therapy | Description, Uses, Examples, & Safety ...

Gene Therapies: Overcoming the Biggest Hurdles in… – Labiotech.eu

By 2024, the gene therapy market is expected to reach an estimated worth of $13B (11.6B). With several therapies approved and hundreds of clinical trials underway, the sector is booming. However, its sudden and fast growth and the complexity of the therapy itself have resulted in a number of hurdles that need to be overcome.

Especially during manufacturing, researchers face challenges at different stages, including process development, safety testing, vector characterization, and when it comes to regulatory guidelines. One of the key issues is the highly compressed timeline researchers have to work with. Instead of the average eight to ten years that it takes to develop a drug, gene therapies are usually developed within three to five years.

Another challenge resulting from the rapid growth of the sector is the increasing demand for plasmids. As the key building blocks for the development of viral vectors, plasmids are needed for gene therapy development. Currently, the industry is struggling to meet the demand for plasmids, forcing companies to think outside the box. This bottleneck has resulted in the development of nonviral vector solutions, which we will see more of in the future.

But compressed timelines and viral vector bottlenecks are not the whole story. This infographic discusses the challenges at various stages of gene therapy manufacturing; what you can do to ace the manufacturing process; and what we can expect in the future.

We developed this infographic in collaboration with Merck. With decades of experience, Merck has already developed three gene therapy products through to commercialization; tested over 10,000 cell and gene therapy samples in one year; and has over 500 batches of different viruses to ensure a smooth manufacturing process.

Author: Larissa Warneck, Science Journalist at Labiotech.eu

Design: Elena Resko

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Gene Therapies: Overcoming the Biggest Hurdles in... - Labiotech.eu

Homology Medicines Announces Peer-Reviewed Publication of HMI-102 Investigational Gene Therapy Demonstrating Restoration of Normal Metabolic Pathway…

- Data Package Supported Initiation of Ongoing pheNIX Clinical Trial forAdults with PKU -

BEDFORD, Mass., March 16, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the peer-reviewed publication of preclinical data that supports Homologys HMI-102 investigational gene therapy program for the treatment of adults with phenylketonuria (PKU). HMI-102 is currently being evaluated in the pheNIX Phase 1/2 clinical trial, and the Company plans to provide an update on the trial when selecting the dose for the expansion part, which is currently anticipated in mid-2020.

The published data shows that a single administration of HMI-102 (AAVHSC15-PAH) produced a sustained reduction in phenylalanine (Phe), the key biomarker in the diagnosis and management of PKU, for the lifespan of the established murine model for PKU. The data also demonstrated a concomitant increase in tyrosine (Tyr), a metabolite of Phe and precursor to neurotransmitters, indicating enzymatic activity. Additionally, brain levels of Phe, 5-HIAA (downstream serotonin metabolite) and coat color were normalized, further indicating restoration of the Phe metabolic pathway.

We developed a robust preclinical data package for our investigational HMI-102 gene therapy, which supported the initiation of our ongoing Phase 1/2 pheNIX clinical trial for adults with PKU, stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. These published data demonstrated that a single dose of HMI-102 was able to restore the normal biochemical pathway in the established PKU model on normal protein diet. Initial data from the pheNIX trial suggests that the increased PAH enzymatic activity after administration of HMI-102 seen in the preclinical model was also observed in the clinical study.

Key data in the publication include:

The publication, Sustained Correction of a Murine Model of Phenylketonuria Following a Single Intravenous Administration of AAVHSC15-PAH, was peer-reviewed and published in the journal Molecular Therapy: Methods & Clinical Development. For more information, please visit http://www.homologymedicines.com/publications.

About the Phase 1/2 pheNIX Clinical Trial in Phenylketonuria (PKU)The pheNIX trial is the first gene therapy clinical trial ever conducted for people with PKU. pheNIX is designed to evaluate the safety and efficacy of a single intravenous administration of HMI-102 in adult patients with PKU aged 18-55. The study design allows for expansion of the number of patients in any dose cohort pending review by the Data Monitoring Committee and the Homology Internal Data Review Team. A decision to expand would trigger the addition of the randomized, concurrently controlled Part B of the trial, which has the potential to be converted to a registrational trial. The primary efficacy endpoint of the expansion part is incidence of sustained plasma Phe concentration 360 mol/L as demonstrated by two measurements 360 mol/L between 16 and 24 weeks.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data from the Phase 1/2 pheNIX trial, including the Part B expansion; plans and timing for the release of clinical data; our beliefs regarding our manufacturing capabilities; advancing our novel platform and pipeline; our goal of delivering potential cures to patients; beliefs about preclinical data; our position as a leader in the development of genetic medicines; the sufficiency of our cash, cash equivalents and short-term investments; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2019and our other filings with theSECcould cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Company Contacts:Theresa McNeelyChief Communications Officer and Patient Advocatetmcneely@homologymedicines.com781-301-7277

Media Contact:Cara MayfieldSenior Director, Patient Advocacy and Corporate Communicationscmayfield@homologymedicines.com781-691-3510

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Homology Medicines Announces Peer-Reviewed Publication of HMI-102 Investigational Gene Therapy Demonstrating Restoration of Normal Metabolic Pathway...

Almost 400 cell and gene therapies in US pipeline, finds report – European Pharmaceutical Review

New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.

A new report from Americas Biopharmaceutical Companies has revealed that there are 362 cell and gene therapies in development in the US. Roughly a third of the therapies, 132, are potential treatments for rare diseases.

The research also highlights that the rate of R&D in this field is growing, as in 2018, a Pharmaceutical Research and Manufacturers of America (PhRMA) report on the cell and gene therapy pipeline found 289 therapies in clinical development in the US.

There are currently nine cell or gene therapy products approved by the US Food and Drug Administration (FDA).

Cell and gene therapies represent two overlapping fields of biomedical research with similar aims, which target DNA or RNA inside or outside the body. Gene therapies use genetic material, or DNA, to alter a patients cells and treat an inherited or acquired disease, whereas cell therapy is the infusion or transplantation of whole cells into a patient for the treatment of an inherited or acquired disease.

According to the report, the novel cell and gene therapies range from early to late stages of clinical development and are focused on a variety of diseases and conditions from cancer, genetic disorders and neurologic conditions.

Some of the cell and gene therapies in the pipeline include:

Another finding highlighted by the report is the 60 RNA therapeutics in development. Whilst not a kind of cell or gene therapy,RNA interference (RNAi) and antisense RNA use a genes DNA sequence to turn it off or modify the gene expression. So, these treatments can potentially inhibit the mechanism of disease-causing proteins.

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Almost 400 cell and gene therapies in US pipeline, finds report - European Pharmaceutical Review

Gene therapy giving Flames executive Snow hope in ALS fight – NHL.com

He swipes once. Twice. Three times.

The lidocaine goes in, to freeze the skin. Then the needle.

Slowly, precisely, Rasquinha removes spinal fluid through the lumbar puncture, commonly known as a spinal tap. He then gives him the injection that Snow and his wife, Kelsie, believe is saving his life.

They believe because they want to, because they have to, and because -- against every single odd, against every single thing you've ever heard regarding amyotrophic lateral sclerosis, or ALS -- it just might be working.

Snow, a 38-year-old assistant general manager for the Calgary Flames, was diagnosed with ALS on June 17. It was not a surprise. His family has been ravaged by the familial form of the disease, with his father, two uncles and a cousin having died from ALS caused by the SOD1 genetic mutation.

ALS affects motor neurons, the cells that control muscle movement. As ALS progresses, the motor neurons die, the muscles become weaker, and eventually movement slows or becomes impossible.

It's what Snow saw happen in his right hand, how he began to suspect that the disease had come for him too. But almost immediately after his diagnosis, which usually carries a life expectancy of between six and 18 months, Snow enrolled in a phase 3 clinical trial at Sunnybrook Health Sciences Centre in Toronto for those with the SOD1 mutation, which affects 2 percent of ALS patients.

In this, he was lucky, both that it was available and that he qualified.

He has made the trip to Toronto with Kelsie every four weeks since then, though initially there was a two-in-three chance that every trip brought him an injection of tofersen, a drug that doctors and researchers hoped could slow the progression of the disease.

For the first six months of the trial, two-thirds of the study participants are given the actual drug. One-third are given a placebo.

He does not know for sure whether he was in the group given the medication or the placebo. The Snows believe he was one of the lucky ones, one of those given tofersen. They believe because they, remarkably, have not seen a progression of the disease since he entered the trial. He still does not have use of his right hand. He has use of everything else, all the things they feared might slip away from him in the weeks and months after they confirmed the diagnosis.

The injection takes two minutes, two minutes of silent meditation for Snow. He thinks about his mom, Linda, who committed suicide in 2012. "Because," as he says, "she'd be really happy and really sad if she were here. One of the things I got from her was a real joy for life. She always was happy that I was that way. So, I think about her and how I get to move on because of this." He thinks about his dad, Bob, who died of ALS in 2018, and his uncles and his cousin.

He wishes they had had the opportunity for these two minutes. For that two-in-three chance.

"I move through those thoughts fairly quickly," he says. "I usually say a prayer or two. For them. Probably in part for myself."

****

The Snows have already been at Sunnybrook for hours at this point, Feb. 20 marking their 11th visit to this hospital, his 10th lumbar puncture. At 9:15 a.m., the driver picks them up at their hotel for the 20-minute ride. When they walk in the doors at the hospital, a massive campus that looks like a small airport, Snow is on edge.

They find their way down to Room UG21, where Snow will undergo a battery of tests, all of them designed to determine whether the progress of the disease has slowed, whether it has stopped, whether it has -- God forbid -- picked up again.

They start with a detailed neurological assessment.

"Have you experienced any changes to your health since you were last here?"

"No."

"Have you noticed any difference in your speech?"

"No."

They test knowledge next, the year, the season, the province, the city. A series of words that never changes from visit to visit: apple, penny, table. He counts backward from 100 by seven.

These are the hardest weeks, when the anxiety sweeps up and the visit looms closer and the fear that "no change" might have turned into "some change" pierces the bubble they have formed around themselves.

"My bad hand, I don't have any expectations for it being better," Snow says. "It's more the opposite, that I'm always kind of concerned about and testing the good parts. The other hand, in particular. Living without one hand is not difficult. Living without two hands is difficult.

"It's testing my good hand, and then I overtire it. And then I convince myself something is wrong."

This is when Kelsie can sense his perpetual optimism faltering.

"None of the physical aspects of any of that, the lidocaine, the needle itself, I couldn't care less about those things," Snow says. "It's just the emotional anguish that you can put yourself through, with wondering, am I a little bit different? And if I'm a little bit different, what does that mean?

"Because that's totally unknown. But then you worry, that could be a slippery slope."

He does a breathing test, to determine lung capacity, and one to test muscle strength. That is the one that gives him the most anxiety, because they are testing his hand and his strength, and that is where the disease started to eat at him.

"This is our 10th time doing most of these tests," Snow says.

"And there has not been any changes," research coordinator Jahan Mookshah says.

"Those are our favorite words," Kelsie says.

Kelsie bends down and ties her husband's shoes. The testing is over, for the moment.

****

There is no blood test for ALS. It's only diagnosed by process of elimination, a factor that often can be problematic, as the disease progresses while the patient is still waiting for answers. Snow was told June 10 by an EMG technician in Calgary that it was likely ALS, a diagnosis that was confirmed a week later by Dr. Michael Benatar at the University of Miami.

It was the last answer they wanted in the world. It was a death sentence.

They crumbled, seeing an unknown present, a future robbed.

"I don't know how we did it," Snow said. "We did our days, and then we cried."

Back in Calgary, fellow assistant general manager Craig Conroy got the news on the phone from Snow, while looking at Snow's kids, Cohen and Willa, now 8 and 5, who were staying at his house while their parents were in Miami. As Conroy said, "That just breaks your heart."

But there was some good news.

Snow had the best-case scenario: a family history and a mutation of the SOD1 gene. He had pressed the issue with doctors, over and over again, after starting to feel the weakness in his right hand while lifting weights during the Stanley Cup Playoffs in April, even as he wanted to believe it was a pinched ulnar nerve, desperate for any answer but this. For Snow, that meant it was only a matter of months between the onset of symptoms and the diagnosis, as opposed to 1 1/2 to 2 years, which is the average.

Which was why when the Snows talked to Benatar on June 17, it was like being flattened and lifted up in the same moment. They confirmed Snow had ALS. They also learned there was a trial, with openings for those with a fast-progressing form of the disease, like him. There was something they could do. There was a place they could go. There was a medicine he could take, produced by Biogen, a company whose corporate headquarters are on Binney Street in Cambridge, Massachusetts, the exact street where Snow lived when he moved there to work at The Boston Globe, another career and another lifetime ago.

There was -- maybe, possibly -- hope. An impossibility in this world.

"It's just like someone believably telling you you're going to die, then telling you you might not die," Kelsie said. "You can't describe that in a more dramatic way than that. You feel like you got your life back. You've got a chance. Just a chance, right?"

Most of the time ALS is sporadic -- that is, not inherited. But in 5 to 10 percent of cases, there is a familial link, an altered gene that has folded. When Kelsie researched trials in the immediate aftermath of the diagnosis, she realized that the studies that were furthest along happened to be for SOD1, Snow's mutation, the second-leading cause of familial ALS.

They would head to Toronto every month, their kids left with a rotating selection of friends, off to get the injection that might -- or might not -- include the liquid hope that his future rested on.

They had six months in which he could have been receiving either the drug or the placebo, until last month, when they were finally assured he would be getting the medication from this point on.

They told their kids that they would have to have a summer to last a lifetime.

"Because we felt like it was maybe going to have to," Kelsie said.

They jammed in everything, all the bike rides and ice cream and pictures, the trip to Merrymeeting Lake in New Hampshire, Snow's family cabin. They jammed happiness in every moment, the tears reserved for private times, for when they couldn't hold it in any longer, wiped away when the kids approached. She wondered if he would make it to February and, if he did, whether he would want to hang around once he got there. She told him to just stay the way he was. They put one foot in front of the other and, still, they don't know exactly how.

By October, they had started to feel differently. Hopeful.

Snow got back on the ice, courtesy of a Flames equipment manager, who sewed his glove into a fist, enabling him to grip a hockey stick. He shot a puck. It rang off the crossbar. Kelsie took a video. It was evident to her that the disease had not progressed, not robbed him of any more strength.

That moment, that month, changed them.

"I felt like that was probably the first moment where I could really breathe," she said.

They went public on Dec. 18, and it ricocheted around the hockey community. Kelsie wrote a letter detailing their diagnosis and their hope, something she has continued to chronicle on her blog, kelsiesnowwrites.com, and recently, in a story that ran in Sports Illustrated.

It has become a piece of her every day, every thought, something that still fells her at a moment's notice. When she is asked if there's been a day that she hasn't thought about ALS since the diagnosis, tears start to flow down her face.

"These three letters are a part of my life forever," Kelsie says. "I sure wish they weren't. But I know that a lot of good things will come to us as a result of this. And I've seen a lot of good in a lot of people because of this.

"There's so much beauty in grief, and there's so much beauty in sadness and tragedy. Because you get to see the best in people. And that's not a small thing. But I wish I didn't have a byline in Sports Illustrated. I don't want a byline in Sports Illustrated. I don't want to be telling this story. But if this is what I'm here for, then I'm OK with that. It's not small. I know that. I know that being there for somebody you love is significant. It's enough for me."

She swipes at the tears, running her fingers underneath her eyes. She sniffles.

"This is because I know you, too," Kelsie says, starting to laugh amidst the tears. "That's the problem."

****

It has been 15 years since I met Kelsie and Chris, back when she was still Kelsie Smith and he was still a baseball writer. The summer of 2005 was one of those summers that's only possible when you are young and there is no responsibility, no ties, no worry. When a White Russian just before the bar closes seems like a good idea, and a shut-off car in a parking lot is the right place to bare a soul, for a friend to reveal she just might be falling in love.

As Kelsie texted me recently, "Honestly, best summer of my life."

They were too young, realistically, for it to work. She was 21. He was 23. They met in a bar, the White Horse Tavern, down the street from the apartment that Snow would buy in Allston, Massachusetts, and that I would later buy from him and live in for the next decade.

He was the Boston Red Sox beat writer at the Globe, she was an intern in the Globe's sports department out of the University of Kansas, and I would be hired into the sports staff that summer as a general assignment reporter.

They got engaged the next summer, and married in December 2007, on a frigid, 9-degree day in St. Paul, Minnesota, where they moved after he was hired as the director of hockey operations for the Minnesota Wild, an unorthodox move that would launch him on a career in hockey and give me a chance to succeed him on the Red Sox beat at the Globe.

She covered the Minnesota Twins for the St. Paul Pioneer Press. We spent spring trainings together in Fort Myers, Florida.

It's hard to think about that day in 2007, so many years ago now, when they promised a lifetime to each other. Because, as Kelsie said, "That I have extra months is not lost on me. I know that I've been already given a gift. It's just that I want what we all thought we were getting when we got married. That we are going to grow old together."

Kelsie has always trusted in Snow, something she has never been shy about expressing. She's always believed he would do what he said, that he would succeed despite all the odds: That he would make the unheard-of jump from baseball reporter to NHL front office member; that he would figure it out after the Wild let him go; that they could find their way on one income (first hers, then his) in a new city, in a new country with a new baby.

And he did. They did.

"It was always like, Chris will figure this out," she said. "And that's probably an unfair amount of pressure that I put on him, but I just believed in him. I've always believed in him that much."

She still does, even in a battle that, up to this point, has been unwinnable.

****

At 1 p.m., after a pizza lunch, it's time to head back to UG21 for the lumbar puncture. Snow laughs about the diet he has been instructed to stick to -- high protein, high fat, high carb -- because losing weight is a marker of the disease. It's one thing he doesn't mind.

This session, too, starts with some tests. Of reflexes. Hands. Ankles. Feet. Jaw.

"This might hurt," Rasquinha says. "Sorry."

Rasquinha flips his hands over, examines them, tells Snow to relax. This is not Snow's forte.

Snow acknowledges yet again that he cannot do anything with his right hand. That went in June, and the atrophy has set in up to his elbow. Kelsie -- or, if he's at work with the Flames, one of his colleagues -- makes sure to cut his food for him, if needed; they tie his skates and his shoes. "Tight, but not too tight," as she puts it.

Testing done, it's time for the lumbar puncture. Snow is now on the open-label extension of the trial. After this visit, he is assured that the fluid sent into his spine will be tofersen. This is a comfort, even as they believe he has been receiving the medication all along.

"All right," Snow says, "let's rock and roll."

The risks are read out, the warnings given, as they always are. The Snows know this nearly by heart. Rasquinha snaps on sterile green gloves and a baby blue face mask. The lidocaine goes in. "Mosquito bite," Rasquinha calls it. "A little burn. Sorry."

Lorne Zinman enters the room. He is part of the reason for their hope, a man who oozes sunshine as he talks, despite having devoted his life to a disease that to this point has claimed every one of its victims.

"This is exciting," says Zinman, the director of the ALS/Neuromuscular Clinic at Sunnybrook and an associate professor of neurology at the University of Toronto. "This is gene therapy for ALS. I've been talking about this for, like, two decades. The fact that it's a reality and we could be helping people like Chris, it's just the happiest thing. I always say -- apology to my children -- the happiest day of my life will be when we finally have something for this disease, because I've seen too many people go through it."

It is easy for Zinman to be joyful these days. He, like all those who work with ALS, has spent years and years in the darkness, and he somehow just might see a little light emerging, a speck that grows bigger with every day that Snow, and potentially others, are stable.

"It's been decades and decades of, really, failure after failure in ALS," Zinman said. "When I give lectures, I put up a slide and I call it the graveyard of failed trials. And it's just failure after failure. The big turning point came when we found the first gene for ALS in the early '90s."

Researchers put the gene into mice, and the mice started to show signs of disease. They thought a cure would come soon after. They were wrong.

Things started to turn when advances in gene therapy led the focus to shift. They knew SOD1 made a protein, and that the protein misfolded, leading to the killing of motor neurons that led to ALS. That became their target.

On Feb. 3, Kelsie posted a video to Twitter showing Snow lifting his right hand at his wrist. It was something he hadn't been able to do since the disease took hold. They don't know what it means, and they try not to read too much into it, but the idea of stability, let alone improvement, sometimes seems like a dream they do not want to end.

And that is what makes Zinman so excited.

"We don't usually see that in ALS trials," he said. "The objective is to slow things down, not to make things improved. It's really exciting when you hear something like that, where I'm actually able to do something that I couldn't do before. You always have to take it with a grain of salt it's just really exciting when you hear something like that."

He envisions a day when a patient like Snow or one of his family members could find out he has the SOD1 mutation, that they could start him on the drugs in the pre-symptomatic phase, as a preventative, similar to the way that doctors have been able to use PrEP to keep HIV from taking hold, or the way that doctors use vaccines to prevent diseases.

"We're diagnosing ALS about five to six times a week, and three to four of our patients die every week," Zinman said. "I've been doing this for 15 years now. I see the faces of these patients, not just them, their family members and what they went through, so to be able to offer someone something like this -- research is hope."

When Zinman and I exited the room, I later learned, Snow grew emotional. He cried. It was joy and relief and release. He had finished another session at Sunnybrook. There had been no change. He soon would be assured of getting more of the medicine that has given him more, where before, he expected there would be less and less.

Because these two minutes, every four weeks, might just be saving him.

"The first few times he was dosed I had full-on panic attacks," Kelsie said. "It was all about the fact that I didn't know what [he was getting]. They'd come in and they'd put this little Ziploc baggie on the desk in there and it had this syringe in it. I remember noticing right away that it was 15 milliliters of this clear fluid. And that was every ounce of hope I had in my life."

****

The appointment over for the month, Snow walks down the corridor, a drab, shabby, beige hallway. It is hardly inspiring, with its fluorescent lights and well-trod flooring. But Snow is nearly bouncing. This is the best he feels all month, his hopes confirmed, the medication, he assumes, flowing through his body.

"Every time I walk this hallway, it's a good feeling," he says. "Because you picture yourself being wheeled."

But he can walk. He can breathe. He can do his job, almost the same way he always has, with a few simple modifications. He wears a suit without a tie these days, because he cannot tie one and, really, who needs it? He has not yet transitioned to slip-on shoes, perhaps out of stubbornness.

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Gene therapy giving Flames executive Snow hope in ALS fight - NHL.com


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