Monthly Archives: September 2022

Biden’s global initiative to replace Christianity with atheism faces …

Posted: September 20, 2022 at 8:55 am

The Biden Administration discreetly launched an anti-Christian globalist grant program in April 2021 that was designed to subvert religious practice and instead ensure "dissent from religious belief" within the "context of intersectional identities."

The program, titled "DRL FY20 IRF Promoting and Defending Religious Freedom Inclusive of Atheist, Humanist, Non-Practicing and Non-Affiliated Individuals," has been called out by congressional Republicans who have noticed that the Biden Administration's defense of "religious freedom" is now "inclusive" of distinctly a-religious disciplines.

According to Fox News, Republican Study Committee (RSC) chairman Jim Banks of Indiana wrote a letter along with 14 other GOP to President Biden and Secretary of State Antony Blinken regarding the Bureau of Democracy, Human Rights, and Labors (DRL) grant program "promoting atheism and 'humanism.'"

In the official letter, Banks noted that atheism "is an integral part of the belief system of Marxism and communism" and that "Americans rightly discern this as a part of the broader effort on the part of [Biden's] administration to promote radical, progressive orthodoxy abroad."

The GOP noted that any program like this within the United States would be unconstitutional and asked "how such a grant or cooperative agreement program advances the foreign policy interests of the United States." The Republicans remarked how foreign leaders could view this program as a type of colonization where America was trying to "shatter local religious and cultural relationships."

The grant program's official website states that is is a Notice of Funding Opportunity (NOFO) that will award one to two grants up to $500,000 through a competitive process to applicant organizations committed to "strengthening networks" and "advocacy" for atheism, humanism, and "non-practicing" religious pluralism in South/Central Asia and in the Middle East/North Africa.

Banks and the GOP wrote, it "is one thing for the Department to be tolerant and respectful of a wide range of belief systems" but "It is quite another for the United States government to work actively to empower atheists, humanists, non-practicing, and non-affiliated in public decision-making."

Banks on Twitter connected the program to Biden's larger radical leftist agenda and tweeted "The Biden State Department is promoting CRT, abortion and now atheism abroad. This is not what America stands for!"

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Researchers to explore why atheism is growing across the world – Brunel University News

Posted: at 8:55 am

An interdisciplinary team of researchers have launched a new project to test popular and academic theories about why some people are atheists and others are not.

Explaining Atheism aims to better understand the growing population of atheists and agnostics in the world, correct inaccurate stereotypes, and give insight into the future of both belief and non-belief.

The project is being led by Dr Jonathan Lanman, Senior Lecturer in Cognitive Anthropology at Queens University Belfast, Dr Aiyana Willard from Brunel University London, and Dr Lois Lee from the University of Kent.

The core research team will investigate the causes of atheism and agnosticism in Brazil, China, Denmark, Japan, the UK, and the USA, with a wider team of affiliated researchers investigating the topic in a further 13 countries across the world.

There are growing numbers of atheists/agnostics in countries across the world, said Dr Lanman. Our recently completed Understanding Unbelief programme looked beyond the stereotypes and helped to document some of the worlds rich diversity in atheism and agnosticism.

Now Explaining Atheism aims to answer the questions of why and how this growth is happening and consider what our answers might mean for the future of religion, atheism, agnosticism, and of our societies.

Dr Lee commented: These are not only academic questions but matters of public debate, policy and law. We are keen to engage the public and the media in our work and we have a funding initiative specifically for those working outside of academia in policy, documentary photography and film, the arts, digital media and data visualisation, education and beyond to help us make sure our work is not only exciting for academics but reaching and learning from wider audiences.

The Explaining Atheism project was awarded 2.7 million in funding by the John Templeton Foundation and will run over a three-year period.

The team launched the Explaining Atheism website which features extensive background information on the project, videos and emerging research findings, with more to come over the course of the project.

Dr Aiyana Willard said: We are excited to launch the Explaining Atheism website. It brings together short films explaining our particular approach to answering these difficult and contentious questions and also provides a number of resources for those looking to explore these questions themselves.

For more information, please visit the Explaining Atheism website http://www.explainingatheism.org and follow on Twitter: @ExplainingAthe1.

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Researchers to explore why atheism is growing across the world - Brunel University News

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Functional atheism: Popular pastor delivers gut-check on what worry really reveals – The Christian Post

Posted: at 8:55 am

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Worry is a normal part of the human experience, though the level to which humans allow it to pervade our minds often exposes unfortunate truths about the state of our hearts.

Pastor Daniel Fusco of Crossroads Community Church in Vancouver, Washington, believes overt fretting is evidence of a deeper spiritual condition.

The problem with worry is worry is functional atheism, he said on his latest episode of the Youre Gonna Make It podcast. When we worry, we act like God isnt in control, [like] Hes not the King of Kings and the Lord of Lords.

READ ALSO: Just Do the Right Thing: Man Sparks Thousands of Reactions After He Finds Wallet in the Middle of Street, Teaches Daughter Powerful Lesson

Fusco continued, We [act like we] dont believe that all things will work together for good.

The preacher is also out with a powerful, new book, Youre Gonna Make It: Unlocking Resilience When Life Is a Mess, in which he discusses this dynamic.

When we choose to trust God, were also abandoning worry, he said.

Fusco knows times have been tough in recent years, highlighting the multitude of challenges stemming from the pandemic and other related crises. Mixed with civil and social unrest, navigating life and culture has perpetuated worries and fear for many.

Thats why Fusco is working to help people steer through times of uncertainty, recognizing God knows we are prone to worry but that we can find peace in Him.

God knows that were apt to worry, Fusco said. God knows that were worry warts.

Listen to him discuss these issues (and subscribe to his podcast):

He said worrying takes a profound amount of energy and that the Lord wants people to instead expend and invest that energy differently.

I can worry about a million things, Fusco said. Whenever I catch myself worrying, I say, Lord, I believe. Help my unbelief I want to turn that worry into worship. I want to invest that energy differently.

Watch Fusco discuss these issues on CBNs Faith vs. Culture:

Fuscos invocation is a profound one, as it comes from Mark 9, where the Bible recounts the story of a demon-possessed boy whose father is desperate for Jesus to heal his son.

Teacher, I brought you my son, who is possessed by a spirit that has robbed him of speech, the man tells Jesus in Mark 9:17 (NIV), continuing in verse 18: Whenever it seizes him, it throws him to the ground. He foams at the mouth, gnashes his teeth, and becomes rigid. I asked your disciples to drive out the spirit, but they could not.

That description alone is enough to instill worry and fear in the calmest of persons. And its clear the despairing father had fear but also hoped Jesus followers and Christ could heal his boy.

But, like many humans, this man seemed to have doubt and worry about whether it would be possible.

READ ALSO: Dire Projections For Christianity in America Over the Next 50 Years Could Have Far-Reaching Consequences For Politics, Family Life, and Civil Society

If you can do anything, take pity on us and help us, the father told Jesus, to which Christ, recognizing this lack of full faith, responded, If you can? Everything is possible for one who believes.

Thats when the grieving dad immediately responded, I do believe; help me overcome my unbelief! (Mark 9:24, NIV).

Jesus then healed the boy. The lesson, of course, is that we can fully trust the Lord and also ask for Him to relieve us of any of the unbelief holding us back.

Find out more about Fuscos new book, Youre Gonna Make It: Unlocking Resilience When Life Is a Mess and subscribe to the podcast.

This story originally appeared on Faithwire.com.

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Chroma Medicine Announces Formation of Scientific Advisory Board of Global Experts in Gene Editing and Cell and Gene Therapy – PR Newswire

Posted: at 8:53 am

Members Bradley Bernstein, M.D., Ph.D., Paula Cannon, Ph.D., Howard Chang, M.D., Ph.D., and Ahmad (Mo) Khalil, Ph.D., will guide advancement of the company's epigenetic editing platform and key programs

Scientific Advisors join Chroma Founders, Luke Gilbert, Ph.D., Keith Joung M.D., Ph.D., David Liu, Ph.D., Angelo Lombardo, Ph.D., Luigi Naldini, M.D., Ph.D., and Jonathan Weissman, Ph.D., expanding the company's world-class team of leaders in genomic medicine

CAMBRIDGE, Mass., Sept 20, 2022 /PRNewswire/ -- Chroma Medicine, Inc., (Chroma) a genomic medicine company pioneering single-dose epigenetic editing therapeutics, today announced the formation of a Scientific Advisory Board (SAB) comprising renowned leaders in epigenetics, cell and gene therapy, and synthetic biology: Bradley Bernstein, M.D., Ph.D., Paula Cannon, Ph.D., Howard Chang, M.D., Ph.D., and Ahmad (Mo) Khalil, Ph.D. The SAB members will provide key input to Chroma as the company advances its programs addressing a wide range of diseases.

"Each of these distinguished experts will be instrumental as we unlock the potential of epigenetic editing therapeutics," said Catherine Stehman-Breen, M.D., Chief Executive Officer of Chroma Medicine. "We are honored to welcome them to the Chroma team and eager to leverage their expertise as we build the future of genomic medicine."

"The SAB is composed of scientific leaders whose seminal research has significantly advanced the fields of genome editing and cell and gene therapy," said Vic Myer, Ph.D., President and Chief Scientific Officer of Chroma. "They bring a wealth of knowledge and experience to Chroma as we continue to advance our platform with the goal of bringing novel single-dose genomic therapeutics to patients."

Members of the Chroma Scientific Advisory Board include:

About Chroma Medicine

Chroma Medicine is a biotechnology company pioneering a new class of genomic medicines that harness epigenetics, nature's innate mechanism for gene regulation, to deliver single-dose therapeutics for patients with genetically driven diseases. The company was founded by the world's foremost experts in genomic research and is led by a veteran team of industry leaders and scientists with deep experience in genomic medicine, drug discovery, and development. For more information, please visit chromamedicine.com or follow the company on LinkedIn and Twitter.

SOURCE Chroma Medicine

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Chroma Medicine Announces Formation of Scientific Advisory Board of Global Experts in Gene Editing and Cell and Gene Therapy - PR Newswire

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Ring Therapeutics Announces Issuance of U.S. Patent for its Anellovector Compositions – Yahoo Finance

Posted: at 8:53 am

Ring Therapeutics

CAMBRIDGE, Mass., Sept. 20, 2022 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize gene therapy with its commensal virome platform, today announced the issuance of U.S. Patent No. 11,446,344. The patent, which was granted to Flagship Pioneering, is exclusively licensed to Ring Therapeutics.

The patent covers anellovirus vectors, which can be used to deliver a diversity of therapeutic modalities. This patent builds on a previous patent (U.S. Pat. No. 11,166,996) granted in November 2021 on methods of delivering a therapeutic polypeptide or nucleic acid by administering Anellovector compositions.

"Ring has comprehensively pioneered a new class of viral vector by generating scientific understanding of anelloviruses through our research work and publications, said Tuyen Ong, MD, MBA, Chief Executive Officer of Ring Therapeutics. This patent issuance supports the novelty of the Anellogy platform and our unique approach of harnessing the human commensal virome to engineer life-saving therapies, ultimately redefining what is possible in programable medicine.

Avak Kahvejian, Ph.D., General Partner of Flagship Pioneering and Co-Founder and Chairman of Ring added, This is another example of Flagship Pioneering companies conducting science at the frontiers of human biology to develop new solutions for humanity. Rings conception of Anellovectors as a broad bioplatform that could be applicable across diseases holds tremendous promise for unlocking the field of genetic medicines.

About Ring Therapeutics

Ring Therapeutics is revolutionizing the gene therapy and nucleic acid medicine space by harnessing the most abundant and diverse member of the human commensal virome, anelloviruses. The company developed the Anellogy platform which focuses on anelloviruses to potentially treat a broad range of diseases. Through harnessing the unique properties of these commensal viruses, the Anellogy platform generates diverse vectors that exhibit both tissue-specific tropism and the potential to be redosed. Founded by Flagship Pioneering in 2017, Ring Therapeutics aims to develop and further expand its portfolio through leveraging its platform to unlock the full potential of gene therapy and nucleic acid medicines, enabling a variety of mechanisms that successfully deliver therapeutic cargo to unreachable organs and tissues. To learn more, visithttps://ringtx.com or follow us on Twitter at@Ring_tx.

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Ring Therapeutics Media:Brittany Leigh, Ph.D.LifeSci Communicationsbleigh@lifescicomms.com+1-813-767-7801

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Cholesterol gene mutation: Why would a healthy 27-year-old have severe heart problems? – 69News WFMZ-TV

Posted: at 8:53 am

An unexplained case of severe coronary artery disease in a seemingly healthy young man led scientists at The Ohio State University Wexner Medical Center and College of Medicine to a discovery that could lead to a new way to treat high cholesterol.

Ohio State scientists discovered rare genetic mutations that had never been identified before, and that could cause severe early onset of coronary artery disease, the most common type of heart disease in the United States. The discovery is leading to a better understanding of how cholesterol functions and the possibility of developing safer therapeutics for the 94 million Americans with high cholesterol.

Marcus Wright, of Delaware, Ohio, was 27 when he suffered a heart attack and continued to have heart-related problems. Doctors were puzzled after finding out he had severe coronary artery disease despite being active and eating a healthy diet.

He was eventually referred to Dr. Ernest Mazzaferri, Jr., an interventional cardiologist at Ohio States Richard M. Ross Heart Hospital and a clinical professor of Internal Medicine.

When I met Marcus about 14 years ago, he looked like a linebacker for the Ohio State Buckeyes. He was super fit and active. He was the last guy that you would ever think would have this kind of disease, said Mazzaferri, co-interim director of Ohio States Heart and Vascular Center. His cholesterol and inflammation numbers looked pretty good and there was no family history of heart disease at that point, so it didn't make sense why somebody like him would have such advanced disease.

Despite optimal medical therapy, Wright continued to have symptoms and needed multiple heart stents, which are expandable metal coils placed in a blocked blood vessel to keep the arteries open.

When both Wrights mother and younger brother were later diagnosed with severe coronary artery disease, Mazzaferri suspected there was a genetic link. He decided it would be a good case for Ohio States JB Project, which is funded by philanthropy and brings together clinicians and scientists to decipher the root cause of highly unusual cases involving coronary artery disease, arrhythmias and heart failure.

Marcus became the first patient I ever presented to our genetic scientists and I said, 'Nobody can figure out what's wrong with this young man. I need your help to understand what's wrong with him,'" said Mazzaferri, who holds the Charles A. Bush, MD Professorship in Cardiovascular Surgery.

A team of researchers led by Sara Koenig, assistant professor of Internal Medicine, conducted genetic sequencing of Wrights DNA and identified unique genetic variants that were causing his advanced disease. To better understand the potential implications of these genetic mutations, Koenig expanded her study, identifying the same mutations in his mother, father and brothers.

This gene encodes a receptor for HDL, which is classically referred to as your good cholesterol, Koenig said. We hypothesize that good cholesterol does not function as well in these individuals as the general population.

The researchers determined that Wrights genetic variants prevented his good cholesterol from effectively clearing out his bad cholesterol (known as LDL), leading to his advanced coronary artery disease. Their findings were published in the American Heart Associations Circulation Research.

The identification of these specific variants in Marcus and his family and knowing that they are causing coronary artery disease sheds lights on a new pathway that we can approach for cholesterol mediated therapy, Koenig said.

Researchers examined 788 FDA drugs approved for various diseases to narrow down which ones may promote a healthy HDL function. They are now working on developing different diagnostic tests and a new therapeutic drug that may provide an alternative option for those living with high cholesterol.

Genetics play a really important role in cholesterol and cardiovascular disease. Even if youre doing everything right by exercising and eating the right foods, some people are just predisposed to high cholesterol and coronary artery disease, which is exactly what happened to Marcus, Koenig said. The issue with statins, which lower your cholesterol, is that theyre not 100% effective. Weve identified a handful of drugs that promote the good HDL pathway or reverse cholesterol transport. Now we are investigating these drugs in animal and human cell models in the hope that we can identify how they affect the pathway and develop more targeted therapies.

Over the years, Mazzaferri has made changes to Wrights medications and now only sees him once a year.

We do the things we do with the hope that we're going to be able to impact people's lives, and to have a group like the JB project work together over a couple year period to really solve a problem like this was one of the most memorable things I'll ever have in my career, Mazzaferri said.

Knowing what was causing his heart problems and learning he probably didnt pass the genetic mutations along to his three children was a relief for Wright.

It was a relief because you're like Okay, now somebody knows something instead of just being a medical mystery, Wright said. They're saying the likelihood is I wouldn't pass it on. As a father, that's the biggest thing that you could be concerned with. I know my parents didn't want to give it to me, and this shouldnt be one of those things that my kids have to worry about.

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Cholesterol gene mutation: Why would a healthy 27-year-old have severe heart problems? - 69News WFMZ-TV

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Gene Therapy for Severe Hemophilia B Could Be More Cost Effective Than Current Treatments – Managed Healthcare Executive

Posted: at 8:52 am

An analysis found that a single dose of gene therapy would be more cost-effective than on-demand or prophylaxis factor replacement in 92% of cases.

Hemophilia is a hereditary, lifelong, and life-threatening disorder that, until recently, has had no prospective cure. Gene therapy might change that, but the cost has been predicted to be in the millions.

Individuals with hemophilia have a gene mutation that causes a deficiency in one of two factors integral in the blood coagulation pathway. In hemophilia A, factor XIII is missing; in hemophilia B, its factor IX. Hallmark symptoms of the disease include excessive bleeding after internal or external trauma. Areas particularly affected are joints, muscles, and soft tissues. In severe cases, bleeding can occur spontaneously.

Current therapy for hemophilia consists of on-demand or prophylactic intravenous infusions of replacement clotting factor to treat or prevent bleeding. Prophylaxis therapy requires several infusions per week, and the cost can range from $300,000 to $776,000 per year.

Recent research on gene therapy introduces the possibility to significantly reduce or eliminate the need for factor replacement therapy. Treatment is designed as a one-time intravenous injection consisting of an adeno-associated virus vector carrying instructions for liver cells to produce deficient coagulation factors.

At least three gene therapies for hemophilia B are currently in late-stage clinical trials, and all have demonstrated at least a 90% reduction in bleeding events and the need for factor replacement. If all goes well, gene therapy can be a potential cure for hemophilia, but the cost has been estimated at $2 to $3 million per patient.

St. Jude Childrens Research Hospital conducted a cost-effectiveness analysis of gene therapy for severe hemophilia B and found that the novel treatment may be more cost-effective than current therapy despite its sky-high price tag. In the analysis, published in the journal Blood last November, St. Jude used its own hospitals data to compare the cost and cost effectiveness of gene therapy with on-demand and prophylaxis factor replacement therapy.

Researchers calculated a total per-patient cost of $87,198 for the manufacturing, distribution, and five-year follow-up of gene therapy for severe hemophilia B. Taking into account the cost of lifelong factor replacement therapy, bleeding complications during on-demand treatments, orthopedic surgery, and hospitalizations, the analysis found that a single dose of gene therapy would be more cost-effective than on-demand or prophylaxis factor replacement in 92% of cases.

According to the authors, gene therapy is in a unique position when it comes to cost analysis. Nancy Bolous, M.D., from St. Jude Global Pediatric Medicine and lead author of the analysis, said, Gene therapy is different because unlike other treatment approaches, it is a long-lasting, one-time treatment and may require a big upfront payment, a payment that can make the affordability seem questionable. Our analysis importance is that it sheds light on the many factors that could play a role in the decision-making process regarding reimbursement.

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AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis…

Posted: at 8:52 am

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AVR-RD-04, an investigational gene therapy for the treatment of cystinosis, a life-threatening disease that causes progressive multi-organ damage, including early, acute kidney disease progressing to end-stage kidney disease.

FDAs Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the company or sold to a third party.

AVR-RD-04 is designed to genetically modify patients own hematopoietic stem cells (HSCs) to express the gene encoding cystinosin, the protein that is critically deficient in people living with cystinosis.

Preliminary data from the ongoing University of California San Diego Phase 1/2 clinical trial suggest that this approach is well tolerated, with no adverse events (AEs) related to the drug product reported to date. All AEs reported were related to myeloablative conditioning, stem cell mobilization, underlying disease or pre-existing conditions. The majority of AEs were mild or moderate and resolved without clinical sequelae. Clinical data to date indicate this investigational approach provides benefits in multiple tissues evaluated, including the eyes, skin, gastrointestinal mucosa and the neurocognitive system. The collaborator-sponsored Phase 1/2 clinical trial is funded in part by grants to University of California San Diego from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).

About CystinosisCystinosis is a rare, progressive disease that impacts approximately 1,600 patients in the U.S., Europe and Japan and is marked by the accumulation of cystine in cellular organelles known as lysosomes. Untreated cystinosis is fatal at an early age. The current SOC for cystinosis, a treatment regimen that can require dozens of pills per day, does not prevent overall disease progression and carries side effects, such as breath and body odor and gastrointestinal symptoms, which can impede compliance. More than 90% of treated cystinosis patients require a kidney transplant in the second or third decade of life.

About AVROBIOOur vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients own hematopoietic stem cells (HSCs), with the goal to durably express the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for cystinosis and Gaucher disease type 1, as well as preclinical programs for Gaucher disease type 3, Hunter syndrome and Pompe disease. Our proprietary plato gene therapy platform is designed to be scaled to support late-stage clinical development and commercialization globally. We are headquartered in Cambridge, Mass. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our preclinical and clinical product candidates, including AVR-RD-04 for the treatment of cystinosis, the potential benefits and incentives provided by FDAs rare pediatric disease designation for AVR-RD-04, the design, commencement, enrollment and timing of planned clinical trials, preclinical or clinical trial results, product approvals and regulatory pathways, our plans and expectations with respect to interactions with regulatory agencies, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our preclinical and investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, including beneficial effects seen in multiple organs and tissues, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our enrollment and development timelines and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis...

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The Biggest CGT Breakthroughs Through the Eyes of Our 2022 Power List – The Medicine Maker

Posted: at 8:52 am

The relatively short history of cell and gene therapy is not lacking in dramatic moments. A previous outlier, this vibrant field now represents the next great hope and so, when roadblocks to progress are removed or even lowered, theres reason to celebrate. Here, seven members of The Medicine Maker Power List 2022, reflect on the most impactful cell and gene milestones.

There have been many significant breakthroughs in cell and gene therapy over the past few years. Specifically in gene-modified cell therapy, the CAR T story is remarkable. Over the past several years, multiple autologous CAR T therapies have been successfully translated from bench to bedside and received marketing authorization as potentially curative therapies for patients with recalcitrant cancer indications: Kymriah and Yescarta for treating r/r/ ALL, MCL, and LBCL, and Abecma for treating r/r multiple myeloma.

Equally impressive in gene therapy, Zolgensma, an AAVSMN1 gene replacement product, has been developed for use as a one-time gene replacement treatment for infants with spinal muscular atrophy (SMA). The 15 year follow-up study these tiny patients are enrolled in after treatment will inform us on the long-term safety and efficacy of gene replacement therapy.

These products have been translated by academia and SMEs and partnered for advanced development with pharma to achieve both medical and commercial success.

The biggest breakthrough is our increasing ability to edit genes with a growing number of new classes of gene editing tools. This advance has led to the boom of CAR T products and is opening the path to cell engineering and in vivo gene therapy.

In parallel, we are seeing an evolution from viral delivery to alternatives with growing payload capacity. This will, as we are already seeing, lead to cures in diseases where that was unthinkable before!

Cell and gene therapies are at the forefront of innovation and transforming how we treat and potentially cure certain diseases. Cell and gene therapies(CGTs) have the potential to treat severe diseases, such as cancer, as well as rare diseases. Several such therapies are now on the market, including a treatment for an inherited retinal disease that causes blindness. That particular CGT represents an important medical milestone because it was the first curative gene therapy approved for use. Personally, I was excited and humbled at the same time to have been the Global Head bringing this transformational therapy to patients around the world. Many other CGTs are now in development and hopefully will lead to an expansion of the still-limited treatment options available to many patients and transform the clinical paradigm.

An important breakthrough? The demonstration that gain-of-function genetically weaponized somatic cells are potent pharmaceuticals in their own right: living synthetic therapeutics (LSTs).Case in point, after a quarter century of work with TILs and LAKs struggling to meet utilitarian endpoints, enter gain-of-function CAR engineering, and thus history is made.The same paradigm of cell gain-of-function genetic enhancement can readily be applied to alternate somatic cell platforms think MSCs and iPSCs with a limitless potential to improve clinical outcomes for acute and chronic ailments.

Id like to emphasize three milestones. First, the commercialization of gene therapies in general. The efficacy and safety have improved a lot since the 1990s.

Secondly, the explosion of immunotherapies. Onco-hematology has become a major opportunity for patients with otherwise lethal blood cancers.

Finally, the advances in gene editing technologies. These have opened the door to new therapies which we would have considered utterly incredible a few years ago.

The recent approval for Yescarta in second-line (2L) relapsed/refractory large B-cell lymphoma (LBCL) means that an order-of-magnitude more patients just became eligible for potentially curative therapies. One recent industry insight from Celltelligence suggested that moving from 3L to 2L will potentially double the targetable population in diffuse LBCL alone for CAR T cell therapy. As cell therapies move up the treatment paradigm and cell-based therapeutics are eventually approved to treat a range of cancers, the spotlight will turn (again) to manufacturing capacity. At Cellares, our belief is that high-throughput, end-to-end automation is set to revolutionize cell therapy manufacturing, allowing us to deliver more doses at lower cost to meet the demand. Its a truly exciting time for our industry!

The success of the CAR T cell therapy approach and how it has led to cures for childhood leukemias and lymphomas is an amazing story. Thanks to these incredible advances, kids who would no longer be here today are now effectively cured, and are going to live long, relatively healthy lives without suffering the long-term side effects of traditional chemotherapy and radiation. By allowing investigators to be highly creative in developing this approach, a fascinating new treatment process was developed, for both autologous and allogeneic CAR T cell therapies. Now, an entire industry has been born from utilizing patients and donors stem cells and a modified version of the AIDS virus to cure leukemia. This is truly a mind-blowing advancement that combines so many complex processes and biologics and really showcases the power of creative investigators to come up with amazing new treatment solutions.

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The Biggest CGT Breakthroughs Through the Eyes of Our 2022 Power List - The Medicine Maker

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Leading Virus Researcher to Chair UVA’s Department of Microbiology, Immunology and Cancer Biology – UVA Health Newsroom

Posted: at 8:52 am

The School of Medicine has named Mariano A. Garcia-Blanco, MD, PhD, as the next chair of its Department of Microbiology, Immunology and Cancer Biology

The School of Medicine has recruited Mariano A. Garcia-Blanco, MD, PhD, an internationally recognized expert in virology and RNA biology, to lead its Department of Microbiology, Immunology and Cancer Biology.

Dr. Garcia-Blanco is a nationally recognized researcher in helping the scientific community better understand gene expression in viruses, and he combines that knowledge with deep experience as both an educator and the leader of a department, said Melina R. Kibbe, MD, the dean of the UVA School of Medicine and chief health affairs officer for UVA Health.Together with this skills and background, he has a clear vision for a department that is conducting cutting-edge research of vital importance.

Garcia-Blanco comes to UVA from the University of Texas Medical Branch, where he has served as chair of the Department of Biochemistry and Molecular Biology since September 2014. A founder of five biotechnology companies and holder of over 11 patents, Garcia-Blanco is a member of the United Nations Council of Scientific Advisers for the International Centre for Genetic Engineering and Biotechnology, and he has previously been a member of the National Institutes of Healths National Advisory General Medical Sciences Council. He has also been elected to the Association of American Physicians and named a fellow of the American Association for the Advancement of Science, the American Academy of Microbiology, and the American Academy of Arts and Sciences.

Continuously funded by the NIH since the early 1990s, Dr. Garcia-Blanco has co-authored more than 190 peer-reviewed scientific publications. His research has focused on how the interactions between proteins and RNA regulate gene expression in cells and viruses. His projects have ranged from examining ways to dial the immune system down in autoimmune diseases and up in cancer to identifying new targets to treat diseases caused by flaviviruses, such as yellow fever, Zika and West Nile. His work has also shed important light on multiple sclerosis and other autoimmune disorders.

As an educator, Dr. Garcia-Blanco has been educating undergraduate, graduate and medical students on topics such as gene regulation, nucleic acids, cancer biology, and autoimmunity, among others.He has mentored over 20 doctoral students and 40 postdoctoral fellows throughout his career and fostered the career development of countless junior faculty.Along with his research and teaching at the University of Texas Medical Branch, he is an adjunct professor of emerging infectious diseases at Duke-NUS Medical School in Singapore. Before coming to Texas, Garcia-Blanco was a faculty member at Duke University from 1990 to 2014.

Our excellent faculty in the Department of Microbiology, Immunology and Cancer Biology will benefit greatly from Dr. Garcia-Blancos inclusive, servant leadership, Kibbe said.I also look forward to seeing how his research continues to help the world better understand both fundamental biology and human disease.

Garcia-Blanco earned his bachelors degree from Harvard University and his MD and PhD from Yale University. He also completed a fellowship at Massachusetts Institute of Technology before joining Duke University.

I am thrilled to be joining the Department of Microbiology, Immunology and Cancer Biology, the School of Medicine, and the University of Virginia, and honored to work with them to achieve excellence in biomedical sciences for the common good, Garcia-Blanco said.

He succeeds Amy Bouton, PhD, who had served as interim chair of the Department since October 1, 2021. Her commitment and dedication to the department has been clear to all and very much appreciated.

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Leading Virus Researcher to Chair UVA's Department of Microbiology, Immunology and Cancer Biology - UVA Health Newsroom

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