FIXX stock is still attracting buyers after a peer-reviewed publication demonstrated the company’s proprietary adeno-associated viral vectors (AAVHSCs) crossed the blood-brain-barrier and blood-nerve-barrier in non-human primates.

Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today a peer-reviewed publication demonstrating its proprietary adeno-associated viral vectors (AAVHSCs) crossed the blood-brain-barrier and blood-nerve-barrier in non-human primates (NHPs), highlighting their potential to deliver gene therapy for central and peripheral nervous system disorders.

The publication includes the initial characterization of biodistribution with three of Homology’s 15 AAVHSCs, including their ability to transduce, or target, key cells following a single intravenous (I.V.) administration in NHPs. AAVHSCs are naturally occurring vectors originally isolated from human hematopoietic stem cells.

“Many neurological diseases, including lysosomal storage and neuromuscular disorders, have cognitive and systemic components requiring a genetic medicine to reach multiple tissues to target the disease-relevant cell types,” said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “Here we evaluated the ability of three of our novel AAVHSCs to cross the blood-brain-barrier and the blood-nerve barrier after I.V. administration in NHPs in addition to other key tissues, which allows us to choose the vectors best suited for particular diseases. We have observed that small sequence changes among our family of AAVHSCs are associated with differences in their ability to target disease-relevant tissues. We continue to characterize these properties and the potential of AAVHSCs as vehicles for therapeutic delivery.”

Following I.V. administration of AAVHSC -7, -15 and -17 in NHPs, analyses showed transduction and transgene expression:

  • Throughout the central nervous system (CNS) in white and grey matter regions as well as the retina, including glial and neuronal cells.
  • In the spinal cord and associated dorsal root ganglia in the peripheral nervous system (PNS), demonstrating the ability to cross the blood-brain-barrier in addition to the blood-nerve-barrier.
  • In peripheral tissues, including the liver, skeletal muscle and heart.

The publication, “Clade F AAVHSCs Cross the Blood Brain Barrier and Transduce the Central Nervous System in Addition to Peripheral Tissues Following Intravenous Administration in Nonhuman Primates,” was peer-reviewed and published in the journal PLOS ONE here: https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0225582

On December 3, 2019, Homology Medicines, Inc. (Nasdaq: FIXX) announced that it has appointed Gabriel M. Cohn, M.D., M.B.A., as Chief Medical Officer. Dr. Cohn will join Homology’s senior management team to direct all clinical development and operations related to Homology’s dual gene therapy and gene editing technology platform. Dr. Cohn’s efforts will focus primarily on advancing the Phase 1/2 pheNIX gene therapy, randomized, concurrently controlled, dose-escalation trial for adults with phenylketonuria (PKU), which is expected to report initial clinical data this year, as well as progress a growing clinical development pipeline.

“Gabe’s expertise in the clinical development of rare disease treatments, including gene therapies and enzyme replacement therapies, comes to Homology at a time when we are advancing our gene therapy trial for adults with PKU,” stated Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “With Gabe on board, we look forward to sharing the initial data from our pheNIX Phase 1/2 trial this month, and to leveraging his experience across multiple genetic disease areas to execute on our programs and expand our rare disease portfolio of potential new treatments and cures for patients in need.”

Dr. Cohn brings to Homology a unique blend of more than twenty years of clinical development experience focused primarily in rare genetic diseases, as well as an academic career focused on reproductive and cancer genetics. Prior to Homology, Dr. Cohn was Vice President, Clinical Development Lead at AVROBIO, Inc., where he led the clinical development for the Gaucher disease and Pompe disease gene therapy programs, providing medical leadership for protocol design, regulatory filings and FDA interactions, global clinical trial site identification and initiation, and investigator interactions. His additional genetic disease experience included positions of increasing responsibility at Shire Plc, including Shire Human Genetic Therapies (HGT), where he most recently held the position of Senior Medical Director, Clinical Sciences, providing strategic medical, scientific and technical expertise and leadership for potential business development opportunities. Before that, he was the interim Global Franchise Medical Lead, Gaucher Disease, focused on developing the franchise strategy, and earlier on served as Medical Director, North America, driving the medical affairs strategy and support for the transition of VPRIV, an enzyme replacement therapy for Gaucher disease type 1, from clinical development through to U.S. launch. Prior to joining industry, Dr. Cohn served as the Chief, Division of Clinical and Reproductive Genetics and Medical Director, Genetic Services at Baystate Medical Center and as Assistant Professor at Tufts University School of Medicine where he won numerous teaching awards. He began his academic career as the Director, Reproductive Genetics at SUNY Health Science Center (HSC) at Syracuse (now Upstate). Dr. Cohn is a Fellow of the American College of Medical Genetics (FACMG), a Fellow of the American Congress of Obstetricians and Gynecologists (FACOG), and author on over 40 peer-reviewed publications, book chapters and review articles, and over 70 published abstracts and presentations. He maintains his medical license in Massachusetts. Dr. Cohn has a Bachelor of Science in Biology from Brooklyn College, a Doctorate of Medicine from SUNY HSC at Syracuse School of Medicine and an M.B.A. from University of Massachusetts Amherst. He completed a residency in Obstetrics & Gynecology at SUNY HSC at Syracuse and a fellowship in Medical Genetics at the National Institutes of Health.

“I have spent my career focused on helping patients, and I am excited to work with the Homology team, many of whom I have worked with before to develop treatments that make a difference in the lives of patients and their families,” stated Dr. Cohn. “I am a strong believer in the potential of Homology’s dual gene therapy and gene editing platform to change the course of rare genetic disease by advancing new treatments and ultimately cures, and I look forward to progressing our clinical pipeline to address the unmet needs of patients.”

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