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Category Archives: Gene Medicine

Advances in the Genetic Etiology of Hearing Impairment – PRNewswire

WASHINGTON, July 19, 2021 /PRNewswire/ --A recently published article in Experimental Biology and Medicine (Volume 246 Issue 13, July, 2021)describes a new genetic mutation linked to hearing impairment. The study, led by Dr. Ambroise Wonkam in the Division of Human Genetics, Faculty of Health Sciences at the University of Cape Town (South Africa), reports a variant of the DMXL2 gene in Cameroonian families with hearing impairment.

The inability to hear properly in one or both ears impacts nearly 6% of the global population. Hearing impairment can be caused by environmental or genetic factors. However, establishing a definitive genetic cause can prove difficult in some cases. Approximately 70% of genetic related hearing impairment cases are non-syndromic and occur without the presence of other clinical factors. Over 120 genes have been linked to non-syndromic hearing impairment.While most cases in Europe and Asia can be traced to variants in a single gene, the GJB2 gene, the etiology of African non-syndromic hearing impairment cases is unresolved.

In this study, Dr. Wonkam and colleagues used direct sequencing methods to analyze DNA samples from a Cameroonian family with non-syndromic hearing impairment (NSHI). A mono-allelic missense variant [NM_015263.5:c.918G>T; p.(Q306H)] was identified in the DMXL2 gene in this family.This variant was present in the heterozygous state in the affected mother and the two affected children (one male and one female), and absent from the other two unaffected children (one male and one female). The variant was absent from many genome databases, over 120 control individuals from Cameroon, and 112 isolated cases of NSHI from Cameroon. This is the first report implicating DMXL2 in NSHI in Africans and confirms a previous report of this variant in China.Dr. Wonkam said, "DMXL2 is now a confirmed NSHI candidate gene in Cameroon, and more studies are needed to assess its implication in other populations around the world."

Dr. Steven R. Goodman, Editor-in-Chief of Experimental Biology & Medicine, said "Dr. Wonkam and colleagues have identified a mono-allelic variant in DMLX2, also called rabconnectin-3a (RC3), in a Cameroonian family with hearing impairment. A similar variant was previously found in a Han Chinese family. It is very interesting that RC3 is found on inner ear hair cells and is a part of a synaptic vesicle protein complex involved in Ca2+-dependent neurotransmitter release in brain. Future studies aimed at a detailed understanding of the role of DMXL2 in hearing impairment is warranted."

Experimental Biology and Medicine is a global journal dedicated to the publication of multidisciplinary and interdisciplinary research in the biomedical sciences. The journal was first established in 1903. Experimental Biology and Medicine is the journal of the Society of Experimental Biology and Medicine. To learn about the benefits of society membership visit If you are interested in publishing in the journal, please visit

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funded study finds gene therapy may restore missing enzyme in rare disease – National Institutes of Health

Media Advisory

Friday, July 16, 2021

Results provide hope for children with aromatic L-amino acid decarboxylase deficiency.

A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare neurological disorder that develops in infancy and leads to near absent levels of certain brain chemicals, serotonin and dopamine, that are critical for movement, behavior, and sleep. Children with the disorder have severe developmental, mood dysfunction including irritability, and motor disabilities including problems with talking and walking as well as sleep disturbances. Worldwide there have been approximately 135 cases of this disease reported.

In the study, led by Krystof Bankiewicz, M.D., Ph.D., professor of neurological surgery at Ohio State College of Medicine in Columbus, and his colleagues, seven children received infusions of the DDC gene that was packaged in an adenovirus for delivery into brain cells. The DDC gene is incorporated into the cells DNA and provides instructions for the cell to make AADC, the enzyme that is necessary to produce serotonin and dopamine. The research team used magnetic resonance imaging to guide the accurate placement of the gene therapy into two specific areas of the midbrain.

Positron emission tomography (PET) scans performed three and 24 months after the surgery revealed that the gene therapy led to the production of dopamine in the deep brain structures involved in motor control. In addition, levels of a dopamine metabolite significantly increased in the spinal fluid.

The therapy resulted in clinical improvement of symptoms. Oculogyric crises, abnormal upward movements of the eyeballs, often with involuntary movements of the head, neck and body, that can last for hours and are a hallmark of the disease, completely went away in 6 of 7 participants. In some of the children, improvement was seen as early as nine days after treatment. One participant continued to experience oculogyric crises, but they were less frequent and severe.

All of the children exhibited improvements in movement and motor function. Following the surgery, parents of a majority of participants reported their children were sleeping better and mood disturbances, including irritability, had improved. Progress was also observed in feeding behavior, the ability to sit independently, and in speaking. Two of the children were able to walk with support within 18 months after receiving the gene therapy.

The gene therapy was well tolerated by all participants and no adverse side effects were reported. At three to four weeks following surgery, all participants exhibited irritability, sleep problems, and involuntary movements, but those effects were temporary. One of the children died unexpectedly seven months after the surgery. The cause of death was unknown but assessed to be due to the underlying primary disease.

Jill Morris, Ph.D., program director, NIHs National Institute of Neurological Disorders and Stroke (NINDS). To arrange an interview, please contact

Pearson TS et al., Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons, Nature Communications, July 12, 2021.

This study was supported by NINDS (R01NS094292, NS073514-01).

The NINDS NINDS is the nations leading funder of research on the brain and nervous system.The mission of NINDS is to seek fundamental knowledge about the brain and nervous system and to use that knowledge to reduce the burden of neurological disease.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit

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Why CRISPR Therapeutics, Editas Medicine, and Beam Therapeutics Dropped This Week – The Motley Fool

What happened

Companies associated with gene-editing are near the end of their second poor week in a row on Wall Street. For the week, shares of CRISPR Therapeutics (NASDAQ:CRSP) were down by 12% as of Thursday's market close. Editas Medicine (NASDAQ:EDIT) was off by about 14% over those four days, and Beam Therapeutics (NASDAQ:BEAM) had lost 15%.

Those downward moves came on the heels of a huge June run-up after Intellia Therapeutics (NASDAQ:NTLA) -- another gene-editing company -- announced that its approach had successfully reversed a genetic disease in human patients. In a clinical trial first, researchers injected a CRISPR treatment into patients that effectively inactivated the body's production of a mutated (and eventually toxic) form of a protein by altering the patients' DNA. Intellia and its partner Regeneron will now navigate the standard regulatory review process. Intellia CEO John Leonard has said he hopes the therapy becomes available to patients "very, very soon." However, marketability could still be years away. Meanwhile, Wall Street's recent surge of excitement about CRISPR therapies has worn off.

BEAM data by YCharts

The drops are notable as investors initially saw this breakthrough result as a positive for all gene-editing stocks. CRISPR Therapeutics, Editas, and Intellia are all taking similar approaches to editing genes -- using the CRISPR-Cas9 enzyme, which functions like a scissors. Beam Therapeutics, on the other hand, uses base-editing, an approach that alters DNA more like a pencil and eraser. Nearly three weeks removed from Intellia's announcement, the market has clearly decided its breakthrough is much more company-specific.

Image source: Getty Images.

It appears gene-editing investors who don't hold Intellia will have to wait for their own companies' catalysts to see big gains. Of these three, CRISPR Therapeutics is the one whose lead candidate is furthest along in clinical trials. CRISPR and its partner, Vertex Pharmaceuticals, have dosed more than 40 patients in a trial studying CTX001 in patients with sickle cell and beta-thalassemia. All patients at least three months removed from the procedure have shown a consistent and positive response to CTX001. Every previously transfusion-dependent patient in the trial has become transfusion-free since receiving the one-time treatment.

CTX001 is currently in a phase 1/2 study, and CRISPR Therapeutics hasn't offered any estimates about when it anticipates that it could be commercially available. But it recently signed an agreement with a smaller startup, Capsida Biotherapeutics, to develop an in vivo therapy for two diseases -- amyotrophic lateral sclerosis (ALS) and Friedreich's ataxia.

Editas has both in vivo and ex vivo (gene-editing done outside the body) candidates in early-stage clinical trials. Its in vivo candidate, EDIT-101, is a treatment for the most common form of childhood blindness. For this program, management has a meeting scheduled with the independent data monitoring committee this summer, and plans to share clinical data by the end of the year.

The company's also developing an ex vivo treatment for sickle cell disease that takes a slightly different approach than the one being used by other gene-editing companies. Editas is using the Cas12a enzyme instead of the more commonly used Cas9. The Cas12a approach has shown better editing efficiency in some studies and only requires one RNA molecule for editing as opposed to Cas9, which requires two.

For now, Beam Therapeutics is furthest back on the research and development path. Its programs are in preclinical stages. Its most advanced candidate also targets sickle cell disease and beta-thalassemia.

Investors' excitement about Beam has been less about its individual treatments and more about the gene-editing technology the company is using. Its base-editing approach could offer a more precise and predictable tool to modify DNA for treating diseases. The company hopes that will allow it to effectively leapfrog its rivals in the next few years. Management has predicted it will file with the FDA for an investigational new drug (IND) designation for its lead candidate later this year. Receiving that designation will give it the green light to test the treatment in humans trials. It also plans to move two more programs into the IND-enabling stage.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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M6P Therapeutics to Host Key Opinion Leader Webinar on Lysosomal Storage Disorders – Business Wire

ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics (M6PT or the Company), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced that it will host a key opinion leader (KOL) webinar on LSDs on Wednesday, July 28, 2021 at 10:00 a.m. ET.

The webinar will feature a fireside chat with KOLs Gregory Enns, M.D., Lucile Salter Packard Childrens Hospital Stanford School of Medicine, and Mark S. Sands, Ph.D., Departments of Medicine and Genetics at Washington University School of Medicine, who will discuss the current treatment landscape and unmet medical needs in LSDs, including Gaucher disease, Fabry disease, Pompe disease, mucopolysaccharidoses, and mucolipidoses. LSDs are a family of approximately 50 rare, genetic, and life-threatening diseases characterized by a deficiency in a specific lysosomal enzyme.

The event will also feature an update from the M6PT management team on its recombinant enzyme and gene therapy S1S3 bicistronic technology platform for the treatment of LSDs. The Company plans to initiate its first clinical program in 2022.

Dr. Enns, Dr. Sands, and M6PT management will also take questions from the audience.

To register for the webinar, please click here.

Dr. Enns is a Professor of Pediatrics and Genetics at the Lucile Salter Packard Childrens Hospital Stanford School of Medicine. He completed his medical education at the University of Glasgow (1990) in Scotland and completed his residency at the Children's Hospital Los Angeles Pediatric Residency in California. He then went on to complete his fellowship at the UCSF Medical Center in California. He is board certified in Clinical Genetics and Genomics. Dr. Enns research interests include novel means of diagnosing and treating mitochondrial disorders, with an emphasis on antioxidant therapy, lysosomal disorders, and newborn screening by tandem mass spectrometry. His current pursuits include the analysis of glutathione and antioxidant status in patients who have mitochondrial disorders and the development of new techniques for diagnosing and treating these conditions.

Dr. Sands is a Professor in the Departments of Medicine and Genetics at Washington University School of Medicine in St. Louis. Dr. Sands received his Ph.D. in Molecular Pharmacology from the State University of New York at Stony Brook. He was a postdoctoral fellow at The Jackson Laboratory (Bar Harbor, ME) and at the University of Pennsylvania School of Veterinary Medicine before joining the faculty at Washington University School of Medicine. The goals of Dr. Sands laboratory are to better understand the underlying pathogenesis and developing effective therapies for inherited childhood diseases, specifically LSDs. A major focus of his group is to determine the safety and efficacy of adeno-associated viral gene transfer vectors for the treatment of both the central nervous system (CNS) and systemic manifestations of these diseases. In addition, his group has developed lentiviral-mediated hematopoietic stem cell-directed gene therapy approaches, as well as small molecule drugs, and more recently rational combinations of these approaches. The primary diseases that Dr. Sands studies are mucopolysaccharidosis type VII (MPS VII), Krabbe disease, and Infantile Neuronal Ceroid Lipofuscinosis.

About M6P Therapeutics

M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary S1S3 bicistronic platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both recombinant enzyme and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its S1S3 bicistronic platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit:

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Leading Gene Writing Company Tessera Therapeutics Announces Pivotal Expansion of Leadership Team – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Tessera Therapeutics, a biotechnology company pioneering a new approach in genetic medicine known as Gene Writing, announced today the appointment of Howard Liang, Ph.D., as President and Chief Financial Officer. The company also expanded its executive bench with newly promoted talent and hires: Madhusudan Peshwa, Ph.D., as Chief Technology Officer for Cell Therapy; Bill Querbes, Ph.D., as Senior Vice President, Therapeutic Discovery & Translational Sciences; Cecilia Cotta-Ramusino, Ph.D. as Senior Vice President, Platform Development; Vikram Ranade, Ph.D., as Senior Vice President, Corporate Development; David Pollard, Ph.D., as Head of Bioprocess, and Steve Garbacz as Head of Finance.

These additions represent the latest leadership expansion for the company, following the appointments of Elliott Sigal, M.D., Ph.D., and Mary Rozenman, Ph.D., to the Board of Directors in June, and the appointments of David Davidson, M.D., as Chief Medical and Development Officer, Hari Pujar, Ph.D., as Chief Operating Officer, and Lin Guey, Ph.D., as Senior Vice President of Rare Diseases Program Strategy and Operations in March. Tessera also announced the successful completion of $230 million Series B financing in January.

Outstanding people are the lifeblood of great companies and Im thrilled to welcome these accomplished individuals to the Tessera leadership team, said Dr. Geoffrey von Maltzahn, CEO and Co-Founder of Tessera and General Partner, Flagship Pioneering. Howards track record in both strategy and finance at BeiGene and in the capital markets will play a key role in guiding Tessera to new territory in Gene Writing. I am excited to be working with him, and our other new senior leaders, each of whom will be instrumental in expanding the limits of how we discover life-changing medicines.

Howard Liang, Ph.D., President and Chief Financial OfficerHoward Liang joined Tessera in 2021 as President and Chief Financial Officer. Dr. Liang brings nearly three decades of combined experience in management, financing, strategy, and research in the biotechnology and pharmaceutical industries and investment research on Wall Street. Prior to joining Tessera, he was Chief Financial Officer and Chief Strategy Officer at BeiGene for six years, where he was a member of the senior team that led the companys growth from a research organization with fewer than 200 employees to a fully integrated global biotechnology company with more than 6,000 employees on five continents. At BeiGene, he led the companys IPOs on NASDAQ and the Hong Kong Stock Exchange and its ongoing effort to list on the Shanghai Stock Exchange, raising more than $8 billion to date through equity and alternative financings, and overseeing the growth of the companys market capitalization from less than $300 million to more than $30 billion during his tenure. Prior to BeiGene, Dr. Liang spent 10 years at Leerink Partners, where he was Managing Director and Head of Biotechnology Equity Research. His prior investment research experience included positions at A.G. Edwards, JMP Securities, and Prudential Securities, covering biotechnology, and major and specialty pharmaceutical sectors. He started his career in R&D at Abbott Laboratories, where he was a Senior Scientist and member of an industry-leading structure-based drug discovery team. Dr. Liang is a member of the Hong Kong Stock Exchange Biotech Advisory Panel. He was named a member of the All-America Research Team by Institutional Investor magazine and Best of the Street by The Wall Street Journal. As a scientist, he authored 14 papers, including 6 in Nature, Science, and Proceedings of the National Academy of Sciences, and a review in the Journal of Molecular Biology. He received his Ph.D. in Biochemistry and Molecular Biology and M.B.A. from the University of Chicago and his B.S. in Chemistry from Peking University.

Tessera is developing a first-of-its-kind technology with the potential to cure diseases across multiple categories by writing in the code of life itself, said Dr. Howard Liang. I look forward to helping the company realize the full breadth of Gene Writings potential.

Madhusudan Peshwa, Ph.D., Chief Technology Officer for Cell TherapyDr. Peshwa joined Tessera in May 2021 and is responsible for developing the strategy and executing the operating plan encompassing the design, development, and manufacture of Tesseras proprietary mobile gene element engineered cell therapy product portfolio. Recently, in March 2020, Dr. Peshwa was inducted into the College of Fellows at the American Institute for Medical and Biological Engineering (AIMBE), in recognition of Lifetime contributions in Regenerative Medicine to the advancements in the field of cell & gene therapies.

Prior to joining Tessera, Dr. Peshwa was CTO at Mana Therapeutics, an immunotherapy company focused on the development of allogeneic, multi-tumor-antigen-targeted, non-engineered, T-cell immunotherapies with additional oversight of Quality Assurance and Quality Control functions. Previously, Dr. Peshwa was CTO and Global Head of R&D for the Cell and Gene Therapies business at GE Healthcare (GEHC), with responsibilities that include GEHCs CGT product and service portfolio to enable and accelerate the development of robust, scalable, industrialized manufacturing and delivery of cell and gene therapies. Prior to these roles, Dr. Peshwa held various executive positions at MaxCyte, Inc., NewNeural LLC, and Dendreon Corporation. At MaxCyte, as CSO and EVP, Cellular Therapies, Dr. Peshwa was responsible for leading the development and commercialization of ex vivo cell loading platform technology. Additionally, he also established MaxCytes proprietary therapeutic product portfolio with lead program being a non-viral mRNA engineered CAR Immunotherapy (CARMA) with one-day manufacturing process under company sponsored IND for treatment of solid cancers; and additional collaborative programs under CRADA Agreement with Investigators at NIAID and NHLBI, for ex vivo gene correction in autologous hematopoietic stem cells, as cell therapy for potential treatment of monogenic diseases. As Vice President of Process Sciences and Manufacturing, at Dendreon Corporation, Dr. Peshwa was responsible for leading the CMC and GMP manufacturing for Provenge (Sipuleucel-T), an autologous cellular immunotherapy product for treatment of prostate cancer, the first ever active cellular immunotherapy product approved by the US FDA.

In addition to his broad industry experience, Dr. Peshwa has served as Principal Investigator / Co-Investigator on multiple grant-funded research studies, is an inventor of six issued US patents in the field of cell therapy, and has served in various consultative, advisory, and board capacities to industry, government, not-for-profit, and financial organizations. Dr. Peshwa earned his Ph.D. in Chemical Engineering from the University of Minnesota and his B.Tech. in Chemical Engineering from the Indian Institute of Technology in Kanpur, India.

Tesseras Gene Writing platform represents an opportunity to drive a fundamental change in our ability to treat disease, said Dr. Madhusudan Peshwa. I look forward to joining the executive team to help move Tesseras bold mission forward.

Bill Querbes, Ph.D., Senior Vice President, Therapeutic Discovery & Translational SciencesBill Querbes joined Tessera in April of 2021 as Senior Vice President of Therapeutic Discovery and Translational Sciences. He brings a strong background in genetic medicine and a passion for rare disease drug development with over 15 years of experience leading cross-functional teams from early discovery through clinical trials.

Before joining Tessera, Dr. Querbes held the position of Vice President and Fabry Program Lead at AVROBIO. Prior to this role, as Senior Director at Synlogic, he led clinical program teams in PKU and urea cycle disorders. Earlier in his career he spent 12 years at Alnylam Pharmaceuticals where he made important contributions to the maturation of both the siRNA delivery platforms and therapeutic pipeline. Dr. Querbes led the discovery and early clinical development of GIVLAARI (givosiran) for the treatment of acute hepatic porphyria, which was the first FDA approved RNAi therapeutic utilizing GalNAc conjugate technology.

He holds a B.S. in Biology from SUNY Geneseo and a Ph.D. from Brown University.

Cecilia Cotta-Ramusino, Ph.D., Senior Vice President, Platform DevelopmentCecilia Cotta-Ramusino joined Tessera in 2019 as the Head of Platform Development. She drives the discovery and optimization of novel Gene Writers, enabling their translation into gene therapy tools. Dr. Cotta-Ramusino has spent more than 20 years in academia and biotech, working in the areas of gene editing, cell engineering, and DNA damage. Dr. Cotta-Ramusino was the first employee at insitro where she was the Head of Functional Genomics. Prior to insitro, she was one of the first scientists hired at Editas, the first CRISPR-based therapeutic company, where she helped to define and shape the vision of the Editas platform. She spearheaded numerous academic collaborations devoted to platform optimization and led the development of a T cell gene therapy treatment aiming to treat an immunodeficiency disease. She conducted her postdoc in Steve Elledges lab at Harvard Medical School where she performed whole genome high-throughput screens in mammalian cells using siRNA/shRNA to identify novel components of the DNA damage response. Dr. Cotta-Ramusino obtained her Ph.D. in genetics at University of Milan, Italy and has been principal author and co-author on several publications in high impact factor journals, such as Science, Nature, Nature Communications and Molecular Cell. She has invented several foundational patents in all of the early-stage companies in which she has worked.

Vikram Ranade, Ph.D., Senior Vice President, Corporate DevelopmentDr. Ranade joined Tessera in 2020 as the Head of Corporate Development. In this role, he drives corporate strategy, business development, and investor relations for Tessera.

Dr. Ranade was previously at McKinsey & Company, where he was an Associate Partner in the healthcare practice. At McKinsey, he worked with large biopharma and early-stage biotech companies on strategy, M&A, and R&D topics. He led diligence efforts for more than $15B in completed deals and advised on clinical strategy for more than 20 programs. Dr, Ranade also co-led McKinseys Center for Asset Optimization, which focuses on clinical-stage asset development strategy. He holds a Ph.D. in Genetics and Development from Columbia University, where he studied transcriptional regulation of developmentally important genes at the molecular level. He has a B.S. in biochemistry from Brandeis University, where he was awarded highest honors for his research on DNA damage repair pathways.

David Pollard, Ph.D., Head of BioprocessDavid Pollard has over 25 years of bioprocess development for a range therapeutics including novel mAbs, peptides, anti infectives, biocatalysts and more recently cell and gene therapies. During his career at Merck & Co. Inc, Dr. Pollard led early and late stage CMC teams, providing contributions to multiple INDs & BLAs for Biologics & Vaccines. Dr. Pollard also led an innovation team that co-developed the state-of-the-art ambr250 high throughput bioreactor system and also pioneered lights out automated continuous mAb production. More recently Dr. Pollard pursued processing for personalized neoantigen T cell therapies and helped create corporate research for the technology provider Sartorius. Dr. Pollard will help Tessera drive digital workflows and high throughput automation to accelerate sustainable gene therapy process development.

Steve Garbacz, Head of FinanceSteve Garbacz joined Tessera in 2021 as the Head of Finance and is responsible for financial reporting, planning, taxes, and treasury. Garbacz has more than 25 years of experience in financial management for a range of companies, including Biogen, Epizyme, Spero, and Anika. He has a passion for building scalable financial organizations leveraging new technology, and drove successful IPOs at Epizyme and Spero. At Anika, Garbacz was a key leader in acquiring and integrating two private companies. Garbacz has a B.S. in Economics from George Mason University and an MBA in Finance from the Leonard Stern School of Business at New York University.

For more information about Tessera, including how Gene Writing works, partnership opportunities, and job openings, visit

About Tessera TherapeuticsTessera Therapeutics is an early-stage life sciences company pioneering Gene Writing, a new biotechnology designed to offer scientists and clinicians the ability to write small and large therapeutic messages into the genome, thereby curing diseases at their source. Gene Writing holds the potential to become a new category in genetic medicine, building upon recent breakthroughs in gene therapy and gene editing while eliminating important limitations in their reach, utilization, and efficacy. Tessera Therapeutics was founded by Flagship Pioneering, a life sciences innovation enterprise that conceives, resources, and develops first-in-category bioplatform companies to transform human health and sustainability.

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Your Healthy Family: New gene therapy providing hope for those with rare genetic disorders – Colorado Springs and Pueblo News

COLUMBUS, OHIO A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. The study, led by researchers at The Ohio State University Wexner Medical Center and The Ohio State University College of Medicine, offers new hope to those living with incurable genetic and neurodegenerative diseases.

Research findings are published online in the journal Nature Communications.

This study describes the findings from the targeted delivery of gene therapy to midbrain to treat a rare deadly neurodevelopmental disorder in children with a neurogenetic disease, aromatic L-amino acid decarboxylase (AADC) deficiency characterized by deficient synthesis of dopamine and serotonin.

Only about 135 children worldwide are known to be missing the enzyme that produces dopamine in the central nervous system, which fuels pathways in the brain responsible for motor function and emotions. Without this enzyme, children lack muscle control, and are usually unable to speak, feed themselves or even hold up their head. They also suffer from seizure-like episodes called oculogyric crises that can last for hours.

Remarkably, these episodes are the first symptom to disappear after gene therapy surgery, and they never return, said study co-author Dr. Krystof Bankiewicz, professor of neurological surgery at Ohio State College of Medicine who leads the Bankiewicz Lab. In the months that follow, many patients experience life-changing improvements. Not only do they begin laughing and have improved mood, but many are able to begin speaking and even walking. They are making up for the time they lost during their abnormal development.

The directed gene therapy in seven children ages 4 to 9 who were infused with the viral vector resulted in dramatic improvement of symptoms, motor function and quality of life. Six children were treated at UCSF Benioff Childrens Hospital in San Francisco and one at Ohio State Wexner Medical Center. This therapeutic modality promises to transform the treatment of AADC deficiency and other similar disorders of the brain in the future, Bankiewicz said.

During the gene therapy surgery, physicians infuse a benign virus programmed with specific DNA into precisely targeted areas of the brain. The infusion is delivered extremely slowly as surgeons monitor exactly how it spreads within the brain using real-time MRI imaging.

Really, what we're doing is introducing a different code to the cell, said Dr. James Brad Elder, director of neurosurgical oncology at Ohio State Wexner Medical Centers Neurological Institute. And we're watching the whole thing happen live. So we continuously repeat the MRI and we can see the infusion blossom within the desired nucleus.

Researchers believe this same method of gene therapy can be used to treat other genetic disorders as well as common neurodegenerative diseases, such as Parkinsons and Alzheimers disease. Clinical trials are underway to test this procedure in others living with debilitating and incurable neurological conditions.

The directed gene therapy, in these patients, resulted in dramatic improvement of symptoms, motor function and quality of life. This therapeutic modality promises to transform the treatment of AADC deficiency and other similar disorders of the brain in the future.

The findings described in this study are the culmination of decades of work by teams from multiple academic institutions, including University of California San Francisco, Washington University in St. Louis, Medical Neurogenetics Laboratory in Atlanta, St. Louis Childrens Hospital and Nationwide Childrens Hospital in Columbus, Ohio.

The research was supported by the National Institute of Neurological Disorders and Stroke and foundational grants, including the AADC Research Trust, the Pediatric Neurotransmitter Disease Association and funding from The Ohio State University.

This work provides a framework for the treatment of other human nervous system genetic diseases. Its our hope that this will be first of many ultra-rare and other neurologic disorders that will be treated with gene therapy in a similar manner, Bankiewicz said.

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