Citrullinemia

Vivet’s VTX-806 is an adeno-associated virus (AAV) vector gene therapy that reinstates CYP27A1 gene activity. Image Credit: Anusorn Nakdee / Shutterstock. The French Government has awarded a €4.9m ($5.3m) grant to Paris-based Vivet Therapeutics to develop VTX-806, a gene therapy to treat a rare condition that leads to the accumulation of cholesterol in nerve cells called cerebrotendinous xanthomatosis (CTX). The funding is part of the France Health Innovation Plan 2023 under the acceleration strategy of Biotherapies – Bioproduction in innovative therapies. The funding is operated by French investment bank Bpifrance and will be disbursed over three years, as per a 1 February press release. Vivet is developing gene therapies for different rare disorders and has a partnership with Pfizer , wherein the latter owns 15% equity in the company. VTX-806 is an adeno-associated virus (AAV) vector gene therapy that reinstates CYP27A1 gene activity. The therapy is being developed to stop or reverse disease progression over the long term, or to potentially cure CTX. CTX is a rare autosomal recessive genetic disorder caused by an abnormality in the CYP27A1 gene. The gene is responsible for producing mitochondrial enzyme sterol 27-hydroxylase enzyme, responsible for converting cholesterol into a bile acid. See Also: Almirall and Microsoft partner for dermatological drug development Takeda and Protagonist sign rusfertide development deal If untreated, CTX can cause progressive neurologic problems such as seizures, cognitive impairment, and ataxia. Current disease treatment includes oral bile acid replacement therapy and treatment with chenodeoxycholic acid to normalise the production of cholestanol. Vivet’s lead candidate, VTX-801, is a gene therapy for Wilson’s disease being developed with Pfizer . The AAV vector gene therapy targets the ATP7B gene, responsible for causing the condition. It is currently being investigated in an open-label Phase I/II GATEWAY trial (NCT04537377), that has a clinical readout expected by the end of the year. The study’s endpoint is to assess the safety and tolerability of VTX-801 at 52 weeks after a single infusion. Additional endpoints include changes in disease-related biomarkers, including free serum copper and serum ceruloplasmin activity, as well as radiocopper-related parameters and VTX-801 responder status to allow standard-of-care withdrawal. Other therapies in Vivet’s preclinical pipeline include VTX-804 for the treatment of a rare neurological disorder, citrullinemia type 1, along with VTX-802 and VTX-803 for the treatment of progressive familial intrahepatic cholestasis (PFIC) – a set of rare genetic disorders. In 2021, US-based Mirum Pharmaceuticals acquired development and commercialisation rights for VTX-802 and VTX-803. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

PHILADELPHIA--( BUSINESS WIRE )-- iECURE , a gene editing company focused on mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need, today announced the appointment of Gabriel M. Cohn, M.D., MBA as Chief Medical Officer. Dr. Cohn will lead iECURE’s clinical strategy for the company’s pipeline of in vivo gene editing therapies, including those designed to treat Ornithine Transcarbamylase (OTC) deficiency, citrullinemia type 1 (CTLN1), and phenylketonuria (PKU). “Gabe’s experience in clinical development as a CMO will be crucial as we begin the process of global regulatory submissions and patient dosing. His diverse experiences in clinical development and gene therapy paired with a robust understanding of genetic disorders and leadership roles in the industry make him the ideal person to take the reins of our expanding clinical team,” said Joseph Truitt, Chief Executive Officer of iECURE. Dr. Cohn brings more than 30 years of experience in academic medicine as well as in the biotechnology industry, where he has contributed to the development of multiple therapeutics for the treatment of rare genetic disorders. Previously, he served as the Chief Medical Officer of Homology Medicines where he led the company’s Phase 1/2 gene therapy trial for PKU and supported two investigational new drug (IND) submissions to U.S. Food and Drug Administration (FDA) for the company’s gene editing programs. Before joining Homology, he served as Vice President of Clinical Development at AVROBIO, leading clinical programs, protocol design, regulatory filings, FDA and Health Canada interactions and trial site identification and initiations. He has also served in executive leadership roles at OvaScience and Shire. Dr. Cohn is a Fellow of the American College of Genetics and Genomics (FACMG) and the American College of Obstetrics and Gynecology (ACOG), author of over 40 peer-reviewed publications, and served as the Chief of Clinical and Reproductive Genetics and Medical Director, Genetic Services at Baystate Medical Center and as Assistant Professor at Tufts University School of Medicine. He earned his M.D. from SUNY Health Science Center at Syracuse and MBA from the Isenberg School of Management at the 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. “It’s a great privilege to join the iECURE team of incredibly successful and seasoned biopharma professionals who have a long track record of developing approved life-altering treatments, while also having an opportunity to collaborate with one of the world’s premier centers of leadership and innovation in the field of genetic therapeutics,” said Dr. Cohn. “They are at the forefront of advancing innovative approaches to gene editing that hold great promise for patients in need, and at this important time of growth and development, I look forward to working with the team and leading our clinical efforts in our goal to successfully treat rare genetic disorders including OTC, CTLN1, and PKU.” About iECURE iECURE is a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need. We believe our approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. We are collaborating with the University of Pennsylvania’s Gene Therapy Program, or GTP, led by James M. Wilson, M.D., Ph.D., to utilize GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of potential product candidates. For more information, visit www.iecure.com and follow on LinkedIn .

Financing co-led by Novo Holdings A/S and LYFE Capital with participation from existing investors Versant Ventures and OrbiMed Advisors Funding expected to enable completion of pre-IND activities, clinical trial initiation, and receipt of clinical data from the Phase 1/2 clinical trial of iECURE’s lead investigational product for Ornithine Transcarbamylase (OTC) deficiency as well as pre-IND activities for Citrullinemia Type 1 (CTLN1) PHILADELPHIA--(BUSINESS WIRE)-- iECURE, a gene editing company focused on the development of mutation-agnostic in vivo gene insertion, or knock-in, editing therapies for the treatment of liver disorders with significant unmet need, announced today the completion of a $65 million Series A-1 financing. The Series A-1 financing was co-led by Novo Holdings A/S and LYFE Capital with significant participation from existing investors Versant Ventures and OrbiMed Advisors. This financing, coupled with the $50 million raised in the prior Series A financing announced in September 2021, will bring the company’s total funds raised to $115 million. As part of the Series A-1 financing, Ray Camahort, Ph.D., Partner at Novo Ventures and Derek Yuan, Ph.D., Managing Director at LYFE Capital will join iECURE’s Board of Directors. Additionally, Tal Zaks, M.D., Ph.D., has been appointed to iECURE’s board as OrbiMed’s new representative on the Board of Directors. Stephen Squinto, Ph.D., will continue to serve on the Board of Directors as an independent member. “In the last year, we have made significant progress in both advancing development of our lead program for neonatal onset OTC and building a world-class team with an extensive track record in developing and commercializing novel therapies,” said Joseph Truitt, Chief Executive Officer of iECURE. “We believe that this funding will enable us to execute all the tasks necessary to begin clinical development of what could be the first mutation-agnostic in vivo gene insertion therapeutic program.” The company expects the proceeds from the Series A-1 financing to enable the advancement of the company’s lead program, GTP-506, including funding IND-enabling studies, starting clinical trials (subject to regulatory approval), and achieving early human data readouts. In addition, the Series A-1 financing is expected to fuel further progress on iECURE’s portfolio of gene editing products for the treatment of patients with rare liver diseases, including citrullinemia type 1 (CTLN1) and phenylketonuria (PKU). “iECURE, through its collaboration with the University of Pennsylvania Gene Therapy Program, has generated impressive pre-clinical data demonstrating durable gene integration in non-human primates with its cutting-edge approach to mutation-agnostic in vivo gene editing. We are excited to work closely with the company as they the move their programs into the clinic,” said Ray Camahort, Ph.D., Partner at Novo Ventures. About GTP-506 iECURE’s approach to gene editing for its initial programs, including OTC deficiency, relies on the delivery of twin adeno-associated virus (AAV) capsids carrying different payloads. GTP-506 comprises two vectors, an ARCUS® nuclease vector (GTP-506A) targeting gene editing in the well-characterized PCSK9 gene locus and a therapeutic donor vector (GTP-506D) that inserts the OTC gene to provide the desired genetic correction. iECURE licensed the ARCUS nuclease for GTP-506 from Precision BioSciences.1 The cut in the PCSK9 site serves as the insertion site for the therapeutic gene, providing a potential path to permanent expression of a healthy gene. The FDA has granted both Orphan Drug Designation and Rare Pediatric Designation to GTP-506 for the treatment of OTC deficiency. About OTC Deficiency OTC deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excessive levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. The only treatment for early onset severe OTC deficiency is a liver transplant. Currently available medical therapies do not correct the disease, and do not eliminate the risk of life-threatening symptoms or crises. About Novo Holdings A/S Novo Holdings A/S is a private limited liability company wholly owned by the Novo Nordisk Foundation. It is the holding company of the Novo Group, comprising Novo Nordisk A/S and Novozymes A/S, and is responsible for managing the Novo Nordisk Foundation’s assets. Novo Holdings is recognized as a leading international life science investor, with a focus on creating long-term value. As a life science investor, Novo Holdings provides seed and venture capital to development-stage companies and takes significant ownership positions in growth and well-established companies. Novo Holdings also manages a broad portfolio of diversified financial assets. Further information: . About LYFE Capital LYFE Capital is a global pioneering healthcare investment platform. We believe that “Healthcare has no boundaries” and that every new choice we made has the potential to advance healthcare and solve the unmet needs for everyone. LYFE Capital leverages our expertise and global resources to invest in and create value for healthcare companies across the globe. Our experienced team has a comprehensive understanding of the healthcare industry across the globe. Management teams value our credibility as we guide them to maximize their potential in a dynamic global market. Further information: . About iECURE iECURE is a gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, editing for the treatment of liver disorders with significant unmet need. We believe our approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit. Our management team has extensive experience in executing global orphan drug and gene therapy clinical trials and successfully commercializing multiple products. We intend to leverage our team’s core strength in research and development strategy to identify what we believe to be the most suitable target and modality for our product candidates to address particular liver diseases. We are collaborating with the University of Pennsylvania’s Gene Therapy Program (GTP) led by James M. Wilson, M.D., Ph.D., to utilize GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of potential product candidates. For more information, visit and follow on LinkedIn. About Precision BioSciences & ARCUS Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (Nasdaq: DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly precise and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company’s pipeline consists of multiple ex vivo “off-the-shelf” CAR T immunotherapy clinical candidates and several in vivo gene editing candidates designed to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit . Penn’s Financial Disclosure The University of Pennsylvania (Penn) and Dr. Wilson each hold equity interests in iECURE. Penn also receives significant sponsored research support from the Company, and both Penn and Dr. Wilson stand to benefit from licensing revenues received from iECURE based on successful technology development and commercialization of the technologies licensed from Penn. Dr. Wilson serves as Chief Scientific Advisor for iECURE. ________________________ 1 iECURE has licensed the ARCUS® nuclease from Precision BioSciences for four gene insertion programs including OTC, CTLN1 and PKU.