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Category Archives: Gene Medicine

GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery – Business Wire

Posted: December 13, 2019 at 3:02 pm

BERKELEY, Calif.--(BUSINESS WIRE)--GenEdit, Inc., a developer of a novel polymer nanoparticle technology platform for non-viral- and non-lipid-based delivery of gene therapies, today announced that it has entered into a worldwide, exclusive license and collaboration agreement with Editas Medicine, Inc., a leading genome editing company. GenEdit has developed a comprehensive delivery system for CRISPR-based therapeutics, including gene knockout and gene repair therapies, to enable safer delivery options with improved efficiency.

"This license and collaboration agreement further validates the strength of our intellectual property portfolio and the potential of GenEdits technology," said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. "We are pleased to establish our relationship with Editas Medicine as they leverage our technology to develop potential genomic medicines."

Under the terms of the agreement, GenEdit has granted Editas Medicine an exclusive worldwide license, with rights to sublicense, to GenEdits Cpf1-based technologies. In return for these rights, GenEdit will receive undisclosed upfront and development milestone payments, including royalties on net sales of products incorporating the licensed intellectual property. In addition, GenEdit and Editas Medicine will collaborate on evaluating delivery of Cpf1-based technologies with GenEdits nanoparticle platform. Editas Medicine will provide research funding and have an option to continue development after the initial collaboration period.

GenEdits nanoparticle platform consists of a proprietary non-viral, non-lipid library of polymers that efficiently encapsulate and deliver cargo [RNA, DNA, protein and/or ribonucleic acid-protein complexes (RNP)] to specific tissues. The company screens the library to identify initial hits and then uses computational analysis and medicinal chemistry for iterative lead optimization. The company has used this platform to identify multiple candidate polymers for efficient and specific delivery of gene editing to a range of tissues.

"Compared to viral vectors and lipid-based nanoparticles, our approach has the potential for better targeting, more cargo, and lower manufacturing cost," said Timothy Fong, Ph.D., chief scientific officer of GenEdit. "In particular, our approach has the potential to enable in vivo gene editing of multiple tissues with CRISPR and expand the potential of gene therapies to treat more diverse sets of diseases."

About GenEdit

GenEdit was founded to transform the delivery of gene and gene editing therapies. We have synthesized the NanoGalaxy library of polymers that can encapsulate RNA, DNA, protein and/or RNP. Through advanced screening methods, computational analysis and iterative medicinal chemistry, we have demonstrated efficient delivery of gene editing cargo to specific tissues. We seek development partnerships for specific tissues and/or gene targets while advancing our internal pipeline of gene editing therapies.

For more information, please visit

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Personalized CF Medicine to be Tested for Rare Genetic Defects in Europe – Cystic Fibrosis News Today

Posted: at 3:02 pm

Three investigative therapies by Proteostasis Therapeutics PTI-801, PTI-808, and PTI-428 showed potential to treat cystic fibrosis (CF) patients ineligible for approved CFTR modulators after patient-specific lab models produced promising results that support a future clinical trial.

Funded by the EUs initiative HIT-CF (Human Individualized Therapy of CF), this early study was conducted on patient organoids, which are miniaturized organ models derived from patient cells.

Based on the positive results, a clinical trial in adult CF patients is expected to start in 2020. If successful, the trial results may serve as the basis for a marketing authorization applicationin Europe in 2021.

With the help of CF-Europe (a patient organization) and the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN), HIT-CF has been recruiting adult CF patients to collect tissue samples and create organoids, three-dimensional organ models grown in the lab.

Organoids mimic several characteristics of the organ from which they are derived, and are genetically identical to the donor. As such, they allow researchers to study biological processes and response to treatments in an environment that closely resembles human organs. As organoids are basically human cells cultured outside the body, they are said to be ex-vivo models.

The idea is to screen treatment candidates in the lab using intestinal organoids, or mini-intestines, obtained from rectal tissue (biopsies) of patients, which are collected through a quick and painless procedure. Then, based on how the organoids respond to each therapeutic candidate, a group of patients will be selected to enroll in a clinical trial.

Because the organoids can help determine the therapeutic candidate most likely to give the best results, researchers can personalize treatments for each patient before starting the trial.

So far, intestinal organoids from more than 300 patients have been created, of which 65 have been used to test Proteostasis potential CFTR modulators.

Proteostasis is honored to have been invited to participate in the HIT-CF project, and is the only company in the group with a combination of novel CFTR modulators being testedex-vivo. We are very enthusiastic about the progress of the study, Geoffrey Gilmartin, MD, chief medical officer of Proteostasis, said in a press release.

The positive results obtained on organoids support the launch of a clinical trial called CHOICES Crossover trial based on HumanOrganoidIndividual response inCF EfficacyStudy that will test the potential treatments on patients whose organoids responded favorably to the agents.

The study will also evaluate if organoids are good models to identify promising CF therapies, that is, if the responses measured on organoids translate to potential clinical benefits in patients, as reflected by lung function tests (FEV1) or sweat tests.

CHOICES will be a placebo-controlled, double-blind study that includes an eight-week treatment period, plus six months of constant dosing.

The study includes Proteostasis three candidate CFTR modulators, specially intended for patients who carry rarer CFTR genetic defects. All three compounds have different modes of action. PTI-801 works as a CFTR corrector, PTI-808 is a CFTR potentiator, and PTI-428 is a CFTR amplifier.

The trial is planned to start in mid-2020, with its first data anticipated for the end of 2020.

If moving forward, CHOICES will become the first trial based on personalized medicine for CF patients.

The inequality in access to CFTR modulators is an acute problem across Europe where 1 in 5 individuals do not have a F508del mutation [the most common CF-causing mutation]. In addition, drug reimbursement policies are leading to an ever-growing gap between patients who do, and those who do not have effective treatment options, said Christiane De Boeck, Work Package Leader at HIT-CF.

At HIT-CF Europe, we believe that novel strategies such as personalized medicine and development of new treatment options are central to addressing the inequality of access across the continent. We are thrilled with these initial results and look forward to providing additional updates, De Boeck added.

Gilmartin said that in Europe more than 2,300 adult CF patients are ineligible for approved CFTR modulators, and therefore excluded from participating in clinical trials.

This projects proposed personalized medicine approach is paving a potential new way to develop and provide access to novel CFTR modulators for patients with the most dire need for treatment options that target the cause of the disease. Additionally, based on an individual patients disease phenotype, and not just the genetic designation, this approach could also create a new path towards more effective treatment for all people with CF, Gilmartin said.

Proteostasis is also running a clinical trial evaluating a combination of the three candidate therapies for the treatment of CF patients who have at least one copy of the F508del mutation in the CFTR gene.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Experts Forum: Creating a cluster of innovation in Rochester – Finance and Commerce

Posted: at 3:02 pm

Click on the image above to see the full magazine.

Innovation being synonymous with Mayo Clinic is nothing new. But now, thanks to Rochesters massive, multiyear undertaking to present the entire city as a beacon for innovators and entrepreneurs, the whole region 75 miles south of the Twin Cities is poised for a technological and economic renaissance.

Finance & Commerce recently hosted a panel discussion about what Rochesters future might hold in those regards. The chat took place at One Discovery Square in Rochester.

The panelists included:

Executive director, Collider Foundation

Traci Downs

President and CEO, Medical Alley Association

Shaye Mandle

Medical director of business development and dermatologic surgeon, Mayo Clinic

Dr. Clark Otley

President and CEO, Vyriad; professor, Mayo Clinic

Dr. Stephen Russell

Director of business development, Discovery Square

Chris Schad

Co-founder and general partner of Capita3

Pam York

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Experts Forum: Creating a cluster of innovation in Rochester - Finance and Commerce

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Sarepta Therapeutics Announces $250 Million of Non-Dilutive Senior Secured Loan Financing – GlobeNewswire

Posted: at 3:02 pm

CAMBRIDGE, Mass., Dec. 13, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that it has entered into an agreement with funds managed by Pharmakon Advisors, LP, that provides Sarepta with up to $500 million of borrowing capacity in two tranches. The first $250 million (Tranche A) is available shortly after closing in December 2019, with an additional $250 million (Tranche B) available at Sareptas option by Dec. 31, 2020, subject to certain conditions. Both tranches are available at a rate of 8.5% annually, payable quarterly. The facility will mature 48 months from the Tranche A closing date. Additional information on the agreement will be set forth in a Form 8-K to be filed by the Company with theSecurities and Exchange Commission.

Pharmakon Advisors, LP, is the investment manager of the BioPharma Credit funds including BioPharma Credit Investments V LP and BioPharma Credit PLC (LON:BPCR), the only listed investor specialized in life sciences debt.

AboutSarepta TherapeuticsSarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit

Forward-Looking StatementThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Sareptas ability to borrow funds under the agreement with Pharmakon Advisors, L.P., and the timing and terms of the borrowing; and Sareptas mission to profoundly improve and extend the lives of patients with rare genetic-based diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Known risk factors include, among others, market conditions, Sareptas market capitalization, any refusal by Pharmakon Advisors, L.P. to fund and other Company factors or circumstances that could negatively impact Sareptas ability to satisfy its obligations or borrow under the terms of the agreement with Pharmakon Advisors, L.P. There can be no assurance that Sarepta will be able to comply with the terms of the agreement with Pharmakon Advisors, L.P., which may result in an event of default under such agreement that could give Pharmakon Advisors, L.P. the right to require immediate payment of any amounts borrowed under such agreement or to exercise its rights with respect to the assets of Sarepta that are collateral or have been pledged by Sarepta as security or other assets of Sarepta; Sarepta may not be able to execute on its business plans, including meeting its expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing its product candidates to market, for various reasons, some of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sareptas product candidates; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2018 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan,

Media:Tracy Sorrentino,

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Care Coordination and Precision Medicine Improve Early Diagnoses –

Posted: at 3:02 pm

December 12, 2019 -Payers are using coordinated care and precision medicine to make diagnoses more quickly and ensure a strong treatment plan for severe and chronic disease management.

Early detection of chronic and severe diseases can mean the difference between life and death. It can also mean the difference between affordable therapies and crippling medical bills.

A March 2018 study found that early cancer diagnosis could result in significant cost savings nationally. Researchers looked at 17 types of cancer and estimated that early detection could save, conservatively, $26 billion nationally.

Recognizing what is at stake, payers take different approaches to catching severe or chronic illnesses in their formative stages.

Coordinated care is a simple, well-tested method for both chronic disease prevention and chronic disease management.

READ MORE: Chronic Disease Coordinated Care May Not Impact Pediatric Spending

Humana recently announced that it would pursue a traditional approach to ensure that patients in danger of chronic kidney and end of life renal disease find out early and get the support they need.

Humana will task skilled provider teams with catching these diseases earlier and implementing personalized treatments.

This coordinated care strategy builds a team of nephrologists, nurses, dietitians, and social workers from one of Humanas two partnerseither Monogram Health or Somatus, depending on geographic location.

The providers will work with the patients primary care physician to determine the best treatments and provide home healthcare options, patient education, and mental healthcare support through counseling.

This multidisciplinary approach will focus on detecting kidney disease earlier, slowing disease progression, and utilizing therapies that enable members to receive care in the convenience of their own home, said William Shrank, MD, MPHS, Humanas chief medical and corporate affairs officer.

READ MORE: Cigna and MSK Start Value-Based, Coordinated Cancer Care Program

Through this collaboration, we will strengthen care coordination for Humana members with kidney disease. Our partnerships will offer customized care options, and will empower patients with education and engagement tools to better manage their condition.

In February, Humana took a similar approach with its oncology program, enhancing its coordinated care strategy and using analytics to ensure quality care.

With new advancements every day in genetic therapies, precision medicine is another method payers use to ensure that patients receive a quick diagnosis and the best treatment plan.

CVS Health launched an oncology care program called Transform Oncology Care, which uses precision medicine to identify and treat cancer patients. The program is rolling out to Aetna members in 12 states but is also available for use by other payers.

Due to CVS Healths geographic and data footprint, it can assess the likelihood that a patient will get cancer. With that information, the patients provider can intervene early on to pursue preventive care, screenings, or therapies.

READ MORE: Precision Medicine Challenges Persist, Aetna Leads Response

When it comes to identifying the appropriate therapies, the program allows providers to use genetics to identify the best course of treatment for a patient recently diagnosed with cancer.

Timing in cancer care is everything and when a patient does not get started on the right treatment it can result in progression and higher costs, said Alan Lotvin, MD, executive vice president and chief transformation officer at CVS Health.

We are the first company working to make the latest in precision medicine accessible to more patients and further empower informed treatment decision-making based on a patient's genetic profile to give them the best chance for successful treatment and improved quality-of-life.

Working in coordination with its third-party vendor, Tempus, CVS Healths new program will enable patients to undergo a broad-panel gene sequencing test once diagnosed to determine the best treatment. This is ideal not only for patients in early stages of cancer, but especially for patients in more advanced stages who need to start treatment as soon as possible.

Because genomic sequencing has certain eligibility requirements, providers are not always aware that gene sequencing is an option open to their patient.

In order to ensure that oncologists prescribe gene sequencing to eligible patients, CVS Health introduced a web-based provider portal into its e-prescribing software which allows oncologists to see the patients eligibility for the broad-panel gene sequencing tests among other functions.

For those who qualify, the program identifies the best treatment options based on genetic makeup. It also alerts providers to potential clinical trials that patients can enroll in and makes the enrollment process easier and faster.

The program integrates National Comprehensive Cancer Network guidelines which are constantly updated for the most recent suggested prescribing and treatment options.

Critically, this service can be employed at the point of detection, so treatments can be identified immediately, and a therapeutic strategy quickly determined.

CVS Health combines this digital solution with a nurse-led coordinated care team to continue quality of care after the diagnosis.

This service is available for only fully insured commercial members.

Among its other chronic disease management developments, earlier this year, CVS Health used preventive care to improve diabetes treatment.

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Aspen Neuroscience Launches With $6.5 Million Seed Funding to Advance First-of-its-Kind Personalized Cell Therapy for Parkinson’s Disease – P&T…

Posted: at 3:02 pm

SAN DIEGO, Dec. 12, 2019 /PRNewswire/ -- Aspen Neuroscience, Inc. today announced its launch following a $6.5 million seed round led by Domain Associates and Axon Ventures and including Alexandria Venture Investments,Arch Venture Partners,OrbiMedand Section 32 to develop the first autologous cell therapies for Parkinson's disease. Aspen's proprietary approach was developed by the company's co-founders, Jeanne F. Loring, Ph.D., Professor Emeritus and founding director of the Center for Regenerative Medicine at The Scripps Research Institute, and Andres Bratt-Leal, Ph.D., a former post-doctoral researcher in Dr. Loring's lab. The company was initially supported by Summit for Stem Cell, a founding partner and non-profit organization which provides a variety of services for people with Parkinson's disease. Aspen is led by industry veteran Howard J. Federoff, M.D., Ph.D., as Chief Executive Officer.

Parkinson's disease is characterized by the loss of specific brain cells that make the chemical dopamine. Without dopamine, nerve cells cannot communicate with muscles and people are left with debilitating motor problems. Aspen is focusing on human pluripotent stem cells, cultured cells that can become any cell type in the human body. The company's research is specific to induced pluripotent stem cells (iPSCs), which it develops by taking a skin biopsy from a person with Parkinson's disease and turning the tissue into pluripotent stem cells using genetic engineering. Aspen then differentiates the pluripotent stem cells into dopamine-releasing neurons that can be transplanted into that same person (autologous), thereby restoring the types of neurons lost in Parkinson's disease.

As an autologous cell therapy for Parkinson's disease, Aspen's treatment would eliminate the need for immunosuppression because the neurons are transplanted back into the same patient from which they were generated. The use of immunosuppression is necessary with currently available cell therapies for Parkinson's disease and when transplanting cells from one patient to another (allogeneic) to prevent rejection but can pre-dispose the patient to life-threatening complications including infection and add cost to the patient and health system. Aspen is the only company in the world offering an autologous neuron replacement therapy for Parkinson's disease.

Aspen encompasses a powerful executive leadership team including Dr. Federoff who, in addition to his leadership roles at the UC Irvine Health System, was the Executive Vice President for Health Sciences and the Executive Dean of Medicine at Georgetown University. Dr. Federoff also has significant biotech industry experience including co-founding MedGenesis Therapeutix and Brain Neurotherapy Bio, as well as leading the U.S. Parkinson's Disease Gene Therapy Study Group. The company is also proud to announce the addition of several experienced and well-known members to its leadership team including Edward Wirth, M.D., Ph.D., as Chief Medical Officer.

Dr. Wirth currently serves as the Chief Medical Ofcer for Lineage Cell Therapeutics where he oversees clinical development of its two therapeutic programs for spinal cord injuries and lung cancer. He received his M.D. and Ph.D. from the University of Florida in 1994 and remained to conduct postdoctoral research including leading the University of Florida team that performed the rst human embryonic spinal cord transplant in the U.S. Dr. Wirth went on to serve as the Medical Director for Regenerative Medicine at Geron Corporation where the world's rst clinical trial of human embryonic stem cell (hESC)-derived product occurred which demonstrated initial clinical safety.

Drs. Federoff and Wirth are joined by Dr. Loring, as Chief Scientific Officer; Jay Sial, as Chief Financial Officer; Andres Bratt-Leal, Ph.D., as Vice President of Research and Development; Thorsten Gorba, Ph.D., as Senior Director of Manufacturing and Naveen M. Krishnan, M.D., M.Phil., as Senior Director of Corporate Development.

"Aspen is developing a restorative, disease modifying autologous neuron therapy for people suffering from Parkinson's disease," said Dr. Federoff. "We are fortunate to have such a high-caliber scientific and medical leadership team to make our treatments a reality. Our cell replacement therapy, which originated in the laboratory of Dr. Jeanne Loring and was later supported by Summit for Stem Cell and its President, Ms. Jenifer Raub, has the potential to release dopamine and reconstruct neural networks where no disease-modifying therapies exist."

Aspen's lead product (ANPD001) is currently undergoing investigational new drug (IND)-enabling studies for the treatment of sporadic Parkinson's disease. Aspen is also developing a gene-edited autologous neuron therapy (ANPD002) that is in the research stage and targeted toward familial forms of Parkinson's disease beginning with the most common genetic variant in the gene encoding glucocerebrosidase (GBA). Aspen leverages proprietary machine-learning tools and artificial intelligence to ensure quality control during manufacturing and to deliver a safe and reproducible product for each cell line.

"Aspen's financial backing, combined with its experienced and proven leadership team, positions it well for future success," said Kim P. Kamdar, Ph.D., Partner at Domain Associates, one of Aspen's seed investors. "Domain prides itself on investing in companies that can translate scientific research into innovative medicines and therapies that make a difference in people's lives. We clearly see Aspen as fitting into that category, as it is the only company using a patient's own cells for replacement therapy in Parkinson's disease."

About Aspen Neuroscience

Aspen Neuroscience Inc. is a development stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinson's disease. Aspen's therapies are based upon the scientific work of world-renowned stem cell scientist, Dr. Jeanne Loring, who has developed a novel method for autologous neuron replacement. For more information and important updates, please visit

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Would you pay $2.1 million for one-off gene therapy? – Kalkine Media

Posted: at 3:02 pm

Glimpse of Health Care Player CSLs Gene Therapy

Wait, What? $2.1 million for just a drug? Yes, you heard it right!

Few months back in Belgium, a fundraising campaign was held to raise funds to pay for a one-time gene-therapy of a toddler suffering from an extremely rare spinal muscular atrophy (SMA)disorder. What has caught everyones attention, was the astonishing $2.1 million price of the drug. The question here is whether it is worth?

What is Gene Therapy?

First lets understand what gene therapy is. Gene therapy is one of the most cutting-edge medical technologies, possessing an untold potential on the brink of revolutionizing the treatment of most debilitating, rare and genetic ailments.

In gene therapy, the expression of a patients genes is modified either by deactivating/blocking or repairing the defective genes, and resuming their normal function, thereby, offering a novel and unique approach for treating life-long and devastating diseases.

One-off gene therapy is a one-time treatment, or a single dose/injection or infusion treatment given to patients.

Prices and One-off Gene Therapy

Although these novel gene therapies present significant advantages to patients with unmet medical needs and have been gaining a lot of praise for their innovative way of disease treatment, but they come at a skyrocket price. For instance,

Such high prices of drugs have ignited debate amid companies (those developed them), stakeholders, and patients.

Payers are still struggling to find out the long-term repercussions of these novel and expensive treatment options that may possibly succeed in addressing and curing disease in just a single dose.

But the companies who have developed these drugs are defending the record-breaking cost of the medicine, arguing that such treatments are still cheaper than the alternate treatment available in the market.

Life-time advantage squeezed down to one-time treatment -Zolgensma Vs Spinzara

There has been a recent fuss in the market when the leading biotech company Novartis joined the debate and defended Zolgensma high price that cures SMA which is a leading cause of mortality in a newborn, emphasizing on the fact that the one-time treatment is more valuable than the costly long-run treatments.

Novartis stated an example of its rival drug Spinraza (an alternative treatment to spinal muscular atrophy, developed by Biogen), which requires infusion in every 4 years at a price of $750,000 in the first year and a maintenance dose of $375,000 per year thereafter.

Me Lennon further emphasized that considering the alternate therapy costs $4 million over a span of ten years, while the one-time cost of Zolgensma is $2.1 million, designating Zolgensma the worlds most expensive drug is deceptive.

Connecting the dots-Reducing Financial Burden

Nevertheless, these novel and costly therapies have intensified the discussions on placing a value on gene therapy and how the government and the health care systems can aid in upfront payment for these one-off treatments.

Shedding light on this, Mr Lennon informed that the company is undergoing discussions with the government bodies and the health insurers for creating new payment models rendering the substantial cost reasonable for payers. With respect to this, a five-year payment plan has already been proposed by the company.

Stakeholders must come up with innovative approaches to price and reimburse the expensive treatment in order to enhance the patient access to these life-changing, novel gene therapies.

Moreover, to realizing the tremendous potential value gene therapies possesses, a reasonable access for all patients is important and so a flexible thinking about evaluating their value.

Key Players

A range of biotech companies have been leading the market leveraging gene therapy approaches while some drugs have entered the pharmaceutical market. Some are listed below-

Let us now have a glimpse of an Australian biotech player that had expanded its technology platform with gene therapy.

CSL limited (ASX: CSL)

Leading Australian Biotech Giant- CSL limited (ASX: CSL) is focused ondeveloping, manufacturing and commercialising novel protein-based pharmaceuticals, cell-culture media & human plasma fractions, with its two key businessesCSL Behring and Seqirus.

CSL has ventured into gene therapy in the fiscal year 2018 after acquiring Calimmune Inc, in 2017 that provide CSL a new technology platform and manufacturing process.

The Company acquired 100% of the Calimmune Incs equity, by making an upfront fee of $82 million and subsequent contingent payments subject to the achievement of development milestones.

Calimmune is a U.S. biotechnology company that has established a suite of gene therapy technologies with a potential to treat rare diseases.

CSLs in-vivo versus ex-vivo cell and gene therapy (Source: Company Presentation)

CSL gene therapy targets Sickle Cell Disease (CSL200), with high unment need and immune deficiencies such as Wiskott-Aldrich Syndrome (WAS).

Two year post the acquisition of Calimmune, CSL has completed the integration of this new technology into R&D with its first clinical program enrolling patients. CSL also has early stage gene therapy projects under pipeline.

On 13 December 2019, CSLs stock traded at $278.12, down 0.38%. The market cap of the company was noted at $126.72 billion with 453.87 million outstanding shares. The stock has a P/E ratio of 46.22x, with 0.95% of annual dividend yield.


This website is a service of Kalkine Media Pty. Ltd. A.C.N. 629 651 672. The website has been prepared for informational purposes only and is not intended to be used as a complete source of information on any particular company. Kalkine Media does not in any way endorse or recommend individuals, products or services that may be discussed on this site. Our publications are NOT a solicitation or recommendation to buy, sell or hold. We are neither licensed nor qualified to provide investment advice.

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The genetic mutation behind a new autoinflammatory disease – Pursuit

Posted: at 3:02 pm

Every minute of every day our bodies are bombarded with millions of different molecules that we breathe, eat and touch including bacteria, viruses, chemicals and seemingly harmless compounds like food and pollen.

For every one of these encounters, our immune system has to decide if the substance is a threat or not, if it is foreign or self and how the body should respond to stay healthy. To do this, we rely on two immune systems working in tandem.

Scientists have discovered a new human autoinflammatory disease that results from a mutation in an important gene in one of these systems.

The syndrome, now known as CRIA (cleavage-resistant RIPK1-induced autoinflammatory) syndrome causes recurring episodes of debilitating and distressing fever and inflammation.

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Our bodys first line of defence is the innate immune system that is effectively a hard wired and fast response, explains Dr Najoua Lalaoui from the Walter and Eliza Hall Institute of Medical Research (WEHI) and the Department of Medical Biology at the University of Melbourne.

This system works in the skin and mucous membranes like the mouth, making sure that any invaders like bacteria are detected and destroyed quickly, she says.

If pathogens do enter the body, the innate immune cells move to the site of infection and physically devour invaders and activate chemical messengers to alert the body.

This can lead to an inflammatory reaction where blood circulation is increased, the affected area becomes swollen and hot, and the person may experience fever. When these chemical messengers are over-active it can result in conditions like colitis, arthritis and psoriasis.

Supporting this system is the adaptive immunity system that involves antibodies that recognise and then train the body to respond to threats. This is our memory immunity and the basis of how vaccinations work.

Scientists from the WEHI, with colleagues at the National Institutes of Health (NIH) in the United States, have been working to understand why patients from three families suffered from a history of painful swollen lymph nodes, fever and inflammation.

The families had a range of other inflammatory symptoms which began in childhood and continued into their adult years.

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This type of repeated fever often indicates an issue with the innate immune system and the same disease in an extended family can indicate genetic changes that are passed from parents to their children, explains Dr Lalaoui.

Previous tests didnt identify any known cause.

But by sequencing the patients genomes, the NIH team identified a mutation in DNA that codes for a molecule known as RIPK that they suspected might cause the disease.

RIPK is a critical regulator of inflammation and the cell death pathway responsible for cleaning up damaged cells or those infected by pathogens.

Professor John Silke from the Walter and Eliza Hall Institute and his team have been studying RIPK1 for more than 10 years. His team had previously shown that damaging the RIPK1 gene could lead to uncontrolled inflammation and cell death.

RIPK1 is a potent controller of cell death, which means cells have had to develop many ways of regulating its activity, Professor Silke says.

In this paper, we showed that one way that the cell regulates its activity is by cleaving RIPK1 into two pieces to disarm the molecule and halt its role in driving inflammation.

In this condition (CRIA), the mutations are preventing the molecule from being cleaved into two pieces, resulting in autoinflammatory disease. This helped confirm that the mutations identified by the NIH researchers were indeed causing the disease, he says.

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He explains that mutations in RIPK1 can drive both too much inflammation as in autoinflammatory and autoimmune diseases and too little inflammation, resulting in immunodeficiency.

There is still a lot to learn about the varied roles of RIPK1 in cell death, and how we can effectively target RIPK1 to treat disease.

In CRIA syndrome, the mutation in RIPK1 overcomes all of the normal checks and balances that exist, resulting in uncontrolled cell death and inflammation, says Dr Steven Boyden from the National Human Genome Research Institute at the NIH.

Dr Boyden says the first clue that the disease was linked to cell death was when they delved into the patients exomes the part of the genome that encodes all of the proteins in the body.

The team sequenced the entire exome of each patient and discovered unique mutations in the exact same amino acid of RIPK1 in each of the three families.

It is remarkable, like lightning striking three times in the same place. Each of the three mutations has the same result it blocks cleavage of RIPK1 which shows how important RIPK1 cleavage is in maintaining the normal function of the cell, says Dr Boyden.

Dr Lalaoui said the WEHI researchers then confirmed the link between the RIPK1 mutations and CRIA syndrome in laboratory models.

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We showed that mice with mutations in the same location in RIPK1 as in the CRIA syndrome patients, had a similar exacerbation of inflammation, she says.

Dr Dan Kastner from NIH widely regarded as the father of autoinflammatory disease says colleagues had treated CRIA syndrome patients with a number of anti-inflammatory medications, including high doses of corticosteroids and biologics, compounds that block specific parts of the immune system.

And although some of the patients markedly improved, others responded less well or had significant side effects.

Understanding the molecular mechanism by which CRIA syndrome causes inflammation provides an opportunity to get right to the root of the problem, Dr Kastner says.

Dr Kastner noted that RIPK1 inhibitors, which are already available on a research basis, may provide a focused, precision medicine approach to treating patients.

RIPK1 inhibitors may be just what the doctor ordered for these patients. The discovery of CRIA syndrome also suggests a possible role for RIPK1 in a broad spectrum of human illnesses, such as colitis, arthritis and psoriasis.

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New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. –

Posted: at 3:02 pm

An American biotech company has launched clinical trials in Colombia to test a new therapy designed to reverse the aging process, and in turn, treat age-related diseases, according to news reports.

But to steal a sip from this purported fountain of youth, participants in the trial must first fork over $1 million a fee that seems even more astronomical when you consider that most clinical trials are either free or provide participants with financial compensation, according to a report by OneZero, a Medium publication about tech and science.

The pricey trial is being run by Libella Gene Therapeutics, a Kansas-based company whose website proclaims that "the future is here." The company announced its intention to test its anti-aging remedies in Cartagena, Colombia, in 2018, and began recruiting for the trials in October of this year. Using a single-gene therapy, Libella aims to "prevent, delay, or even reverse" the general effects of aging, as well as treat diseases that emerge in old age, such as Alzheimer's, according to

In fact, in its own press release, the company boasted, without evidence, that its gene therapy "may be the world's first cure for Alzheimer's disease." The bold claim raises an obvious question: Will the treatment actually work?

Short answer: No one really knows, but the fact that Libella shipped its operation beyond the reach of the U.S. Food and Drug Administration (FDA) doesn't inspire confidence, experts told OneZero.

Related: 5 Reasons Not to Fear Getting Older

Unlike anti-aging face creams that soften the superficial signs of aging, the Libella therapy aims to reverse aging from the ground up, so to speak, starting at the level of our genes. Specifically, the gene therapy is intended to lengthen patients' telomeres structures that cap the tips of chromosomes and prevent the genetic material inside from fraying. Telomeres grow shorter each time a cell divides, and when the structures reach a critical length, cells either stop dividing or perish, according to Stanford Medicine.

The theory goes, if you rebuild the body's shortened telomeres, the process of aging might be thrown in reverse. This is not a new idea. Several studies in mice suggest that using gene therapy to lengthen telomeres can reverse certain signs of aging in the animals. A 2015 study from Stanford prompted similar effects in isolated human cells; the treatment lengthened cells' telomeres by fiddling with a close cousin of DNA, called RNA, which helps cells build proteins.

The Libella therapy aims to help cells rebuild telomeres by activating a gene in their DNA that would normally be switched "off." The gene, called TERT, contains instructions to build a protein called "telomerase," an enzyme that adds molecules to the end of telomeres and prevents the structures from shortening during cell replication, according to a 2010 report in the journal Biochemistry.

Libella's lead scientific officer, molecular biologist William Andrews, originally helped identify the human telomerase enzyme at the biotech firm Geron. Later, he licensed a gene therapy based on the finding to Libella, according to OneZero. "I can't say [telomere shortening is] the only cause of aging, but it plays a role in humans," Andrews told the publication.

Related: 8 Tips for Healthy Aging

Andrews' therapies will soon be put to the test in Colombia, where one 79-year-old will receive the anti-aging treatment in next month, according to OneZero. The anti-aging trial will include four more participants over age 45 and focus on verifying that the treatment is "safe and tolerable," meaning it does not harm patients or cause unacceptable side effects.

Two more trials will use the same therapy but aim to "prevent, delay, or even reverse the development" of Alzheimer's disease and critical limb ischemia, an age-related condition in which a person's arteries become severely obstructed. Participants in these trials must already be diagnosed with the disorders.

After treatment, participants in all three trials will remain in the clinic for 10 days for further monitoring, and then return at regular intervals for checkups over the following year.

Libella's gene therapy involves a one-time injection delivered through an IV; the Alzheimer's therapy uses the same formula but doctors inject the product into the patient's spinal fluid. Within the product, a modified virus carries the TERT gene into cells and injects the genetic material into their DNA. The modified viruses cannot transmit diseases to people, but in high enough doses, the germs could provoke a harmful immune response in the patient, according to a 2018 animal study. Libella representatives declined to say how high a dose their clinical trial participants will receive.

"All I can say is, it's a lot," Andrews told OneZero.

Potential side effects aside, the fact that the Libella treatment will be administered beyond the purview of the FDA is telling, according to one expert. Leigh Turner, a bioethicist at the University of Minnesota, told OneZero that "even though the company is based in the United States, they've managed to find a way to evade U.S. federal law by going to a jurisdiction where it's easier to engage in this activity."

The $1 million entry fee is also alarming, Turner said, given that most clinical trials don't charge patients anything to enter. Andrews told OneZero that the fee is justified because it costs the company hundreds of thousands of dollars to make enough product to treat just one person.

The appearance of the trials on, an official registry maintained by the National Institutes of Health, does not boost their credibility, she added. The automated database can be easily manipulated and "can basically be used as a marketing platform," she said.

Other stakeholders in the telomere-lengthening business are concerned, too. Michael Fossel, founder and president of the biotech startup Telocyte, told OneZero that his company's own therapy is similar to the Libella treatment the difference is that Telocyte is seeking approval through the FDA. "We're afraid that something will go wrong [with the Libella trials], whether it's from a safety or efficacy standpoint," he said.

Related: Extending Life: 7 Ways to Live Past 100

But even in a best case scenario, wherein no patients come to harm, the Libella therapy still might not deliver any notable health benefits. Some research suggests that no link exists between telomere length and aging.

For instance, a study published this year examined more than 261,000 people between age 60 and 70, and found no correlation between participants' telomere lengths and their age-related health outcomes, including their overall cognitive function, muscular integrity and the age of their parents. Long telomeres were associated with a lowered risk of coronary heart disease as compared with short telomeres, but longer telomere length was also linked to a heightened risk of cancer.

"Telomere lengthening may offer little gain in laterlife health status" and lead to an increased risk of cancer, the authors noted.

It remains to be seen whether Libella has truly tapped the fountain of youth, but given the dubious nature of their clinical trials, potential participants may want to exercise caution before relocating to Colombia and shelling out $1 million for a chance to live longer.

Originally published on Live Science.

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Updates from SABCS 2019: Detection and Screening, Immunotherapy Advances, and Therapy Resistance – On Cancer – Memorial Sloan Kettering

Posted: at 3:02 pm

At the annual San Antonio Breast Cancer Symposium, MSK investigators presented the latest research on detection and screening methods for people at high risk;immunotherapy for breast cancer;and the underlying causes of resistance to targeted therapies, among other topics.

Here are some of the noteworthy studies that featured contributions from MSK investigators.

Mammography screening has been shown to reduce breast cancer mortality by about 30% in the general population. But in women at an increased risk for the disease, additional imaging is recommended. This group includes people who carry a BRCA or other genetic mutation. Other risk factors include a family or personal history of breast cancer, certain high-risk lesions, or having undergone chest radiation at a young age.

At SABCS, diagnostic radiologist Maxine Jochelson discussed newer imaging technologies and the advantages they have over mammograms alone for detecting cancer in high-risk women. People in the high-risk group may need supplemental imaging to improve early detection, Dr. Jochelson says.

She explains that this approach would incorporate vascular imaging techniques. These methods can highlight areas of increased blood flow, a hallmark of tumor growth. This technology includes MRI and contrast-enhanced mammography. It can find tumors that mammograms may miss. Although vascular imaging costs more and generally takes longer to perform, its use is justified in high-risk women because ofthe increased chance of finding cancer, she says.

Mammograms & Other Types of Breast Exams

Learn about the different types of breast exams that can help detect breast cancer at its earliest stages, before symptoms develop.

Its undisputed that vascular imaging is better at detecting cancers than purely anatomical imaging, Dr. Jochelson adds. She emphasizes the need to fine-tune imaging strategies based on each persons specific risk factors.

Some of the imaging approaches she discussed during her presentation include:

We need to continue improving ways of assessing an individuals risk so we can stratify them and determine which type of imaging will most benefit each patient, Dr. Jochelson says. The true test will be studies to demonstrate that these newer technologies actually save lives.

Immunotherapy that uses genetically engineered cells, such as chimeric antigen receptor (CAR) T cells, has proven effective in treating some forms of blood cancer. So far, efforts to create immune cells that can effectively target solid tumors, including breast cancer, have been disappointing. At SABCS, MSK physician-scientist Christopher Klebanoff presented research from his lab on a novel tactic for enabling the immune system to better target and kill breast cancer cells while sparing healthy tissue.

We believe a major limiting challenge in successfully developing immunotherapy for breast cancer has been the identification of antigens. These are targets that the immune system can recognize, Dr. Klebanoff explains. Weve become very interested in the possibility that common mutations in breast cancer may produce antigens that can be recognized as foreign by the immune system.

The Klebanoff labs current research focuses on a gene called PIK3CA, which is mutated in about 40 to 45% of hormone receptor-positive breast cancers. It is also mutated in some HER2-positive and triple-negative breast cancers. Mutations inPIK3CA cause cancer cells to grow in an uncontrolled manner. In May 2019, the US Food and Drug Administration approved a pill called alpelisib (Piqray), which targets mutations in this gene. However, the drug has the potential for significant side effects, and tumors ultimately develop resistance to this medicine. Dr. Klebanoff and his colleague Smita Chandran, a senior research scientist in his lab and the scientific lead on this study, decided to look for a way to target antigens created by this mutation using immune cells designed to recognize them.

We believe a major limiting challenge in successfully developing immunotherapy for breast cancer has been the identification of antigens.

A challenging aspect of this approach was that mutated PIK3CA is found on the inside of cancer cells, allowing it to hide from many components of the immune system, such as antibodies. Physiological processes present in all cells, including cancer cells, allow mutated PIK3CA to be broken down into shorter fragments and loaded onto a molecular basket, called HLA, which is shuttled to the surface of the cell, Dr. Klebanoff says. This process allows immune cells to functionally look inside of other cells.

The researchers identified a specialized molecule, known as a T cell receptor, that has the ability to recognize this mutated PIK3CA-HLA complex. Immune cells specific for this complex recognize the target cell as being cancerous and destroy it. Healthy cells without the mutation remain untouched. The T cell receptors are matched to a patients unique complement of HLA molecules. As with a stem cell transplant, HLA must be matched for this immunotherapy to be effective.

Right now we are focused on the most common HLA types that are seen in a large proportion of our patients. The big-picture goal is to build a library of T cell receptors that can work in people with different HLA molecules and can target other common cancer mutations, Dr. Chandran explains. This work is still early and so far has only been done in the laboratory and not in humans. We are nonetheless excited about the prospect of working toward developing a more effective and less toxic immunotherapy customized to the genetic attributes of a patients tumor.

CDK4/6 inhibitors are an important class of drugs to treat estrogen receptor-positive breast cancer. These drugs stop the growth of breast cancer cells by targeting enzymes that are important in cell division. They are given in addition to hormone therapy. But about 10 to 15% of people who get these drugs dont respond to CDK4/6 inhibitors, and others later develop resistance.

MSK physician-scientist Sarat Chandarlapaty has been studying why this is the case. Understanding this resistance could contribute to the development of new targeted drugs. In December 2018, he published a study that reported on two genes that play a critical role in promoting this resistance. At SABCS, he presented his latest research on this area.

Weve been delving deeper into the role of these genes, as well as others, to try to understand some of the principles that could guide the next generation of therapies, Dr. Chandarlapaty says. By working out these detailed mechanisms, we will have the tools needed to design more potent and selective inhibitors for these refractory breast cancers.

Dr. Chandarlapaty explains that because tumors outsmart CDK4/6 inhibitors in different ways, he doesnt expect to find a one-size-fits-all approach for new drugs. There are some key principles for why these drugs fail, he says. For some tumors, making a more potent drug of the same general class will work. Other tumors bypass the pathway in a way that renders many of the old therapies weve used ineffective. For them, a completely different approach is needed.

Researchers Identify Why Women May Develop Resistance to a New Class of Breast Cancer Drugs

Clues emerge about why promising new breast cancer drugs sometimes dont work and what might be done about it.

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Updates from SABCS 2019: Detection and Screening, Immunotherapy Advances, and Therapy Resistance - On Cancer - Memorial Sloan Kettering

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