BBS1

Viralgen will manufacture the therapy at its facility. Credit: Viralgen/PRNewswire. Viralgen and Axovia Therapeutics are set to form a partnership to progress the development and manufacturing of an adeno-associated virus vector serotype 9 (AAV9)-based gene therapy for retinal dystrophy in individuals with Bardet-Biedl Syndrome (BBS) due to BBS1 gene mutations. The collaboration seeks to prevent vision loss in BBS patients. The investigational gene therapy utilises a codon-optimised BBS1 AAV9 vector tailored to slow down vision loss caused by genetic defects. Viralgen will manufacture the therapy at its facility, employing its expertise in AAV and the Pro10 cell line and manufacturing platform. Combined with Axovia’s gene therapy development knowledge, the company seeks swift delivery. Axovia co-founder and chief scientific officer Dr Victor Hernandez stated: “As Axovia advances its pipeline of potential therapies addressing the genetic causes of blindness towards the clinic, we want to ensure we have appropriately scaled AAV manufacturing to support this effort, and are very happy to have partnered with Viralgen as our CDMO [contract development and managing organisation]. “Our lead programme, AXV-101, which is being developed to address retinal dystrophy associated with BBS, is expected to enter clinical development in mid-2025, and we believe this partnership will ensure fast and efficient development as we seek to advance this therapy toward patients as soon as possible.” BBS leads to multi-system impairments, including obesity and progressive retinal dystrophy. Viralgen, established in 2017 as AskBio’s independently operated subsidiary, is part of the Bayer Group. The company’s facilities in San Sebastián, Spain , feature a commercial facility with three modules, each housing three current good manufacturing practices suites with capacities exceeding 2,000L. 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. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, 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

PHOENIX, Oct. 30, 2024 (GLOBE NEWSWIRE) -- A Race Against Blindness, a nonprofit organization dedicated to funding sight-saving research for children, is pleased to announce a $1 million grant to Axovia Therapeutics to support the company’s lead program, AXV-101. This marks an important step toward developing a treatment to address blindness and obesity associated with Bardet-Biedl Syndrome 1 (BBS1), a rare genetic disorder with no current curative options. The funding from A Race Against Blindness will directly support Axovia's near-term milestones associated with the initiation of the clinical study and initial patient enrollment in the United Kingdom in mid-2025 to treat blindness caused by BBS1 mutations. Axovia Therapeutics’ future plans include initiation of a clinical trial in the United States and progression of the program to also treat obesity. BBS is a devastating genetic disorder associated with primary cilia dysfunction. One of the hallmarks is retinal degeneration commonly presenting as retinitis pigmentosa. The other hallmark is hyperphagia resulting in morbid obesity, among other complex clinical manifestations. Of the many genes known to be associated with BBS, pathogenic variants in the BBS1 gene are most commonly encountered. Axovia is developing a genetic therapy for those harboring two mutations in the BBS1 gene. There is no cure for BBS, and its prevalence ranges between one in 70,000 and one in 100,000 in Europe and North America, with up to 10 times this prevalence in the Middle East. “Our $1 million grant to Axovia is a significant step for the BBS community, and we are optimistic about Axovia’s groundbreaking clinical research. However, the path to treating RP/BBS is challenging and there is still much work to be done. A Race Against Blindness is committed to supporting the effort to raise the necessary funds to ensure the execution of the AXV-101 clinical trial and potentially bring a new therapeutic option to BBS patients like our son, Luke,” said Dr. Stephen Johnston, Co-Founder and President. “We are advancing the AXV-101 program and preparing to initiate the first clinical study in mid-2025. We are immensely grateful for this funding from A Race Against Blindness,” said Professor Phil Beales, Chief Executive Officer and Co-Founder at Axovia Therapeutics. “By utilizing gene therapy to address the loss of sight and obesity experienced by patients with BBS1, we believe our approach will have a profound and dramatic impact on the health and well-being of this community. We look forward to generating clinical data for this program.” With the goal of initiating clinical studies in mid-2025, A Race Against Blindness will continue aggressive efforts to fundraise, engage the scientific community, and raise global awareness of BBS. Media Contact:Stephen Johnston, M.D., Co-Founder and PresidentSteve@ARaceAgainstBlindness.org (520) 548 - 1807

Developing the first gene therapy to treat diseases caused by cilia dysfunctionAXV101 targets retinal dystrophy for Bardet-Biedl Syndrome (BBS) patientsFDA Orphan Drug Designation and Rare Pediatric Disease Designation achievedFirst-in-man study planned to start in late 2024/early 2025Six potentially transformative therapies for ciliopathies are in its pipeline LONDON, Sept. 19, 2023 (GLOBE NEWSWIRE) -- ALSA Ventures, a UK-based, therapeutics-focused venture capital fund, announces the acquisition of Axovia Therapeutics Inc and the launch of a new portfolio company Axovia Therapeutics Ltd. Axovia is developing the first gene therapies for ciliopathies and has a pipeline of products for these devastating diseases, including Bardet-Biedl Syndrome (BBS). The ALSA Ventures’ investment team has designed an accelerated development plan to take the lead program AXV101 into clinical trials in the next 18-24 months with a rapid path to clinical proof of concept and approval. AXV101 is an AAV9-based gene therapy targeting retinal dystrophy associated with BBS in patients carrying biallelic mutations in the BBS1 gene. It is designed to halt retinal degeneration, which begins in childhood leading to blindness before 20 years of age. Revised epidemiological analyses suggest that BBS affects between 1 in 70,000 - 1 in 100,000 in Europe and North America, and there is no treatment for the retinal degeneration. ALSA Ventures CEO, Alek Safarian, said they were excited to take AXV101 into clinical trials, which will have access to well-characterised and motivated patients. “Especially pediatric patients who need early treatment before permanent vision damage occurs,” he said. “Our investment team was particularly attracted to Axovia Therapeutics as a valuable addition to our growing portfolio of companies because of its striking preclinical results, its Rare Pediatric Disease Designation, and the proven AAV delivery mechanism,” he said. The company is well positioned for an FDA priority review voucher (PRV) which is awarded to sponsors that develop drugs for diseases, including rare paediatric diseases. Axovia is based on decades of work on ciliopathies at University College London by co-founders Professor Phil Beales and Dr Victor Hernandez. Axovia Acting CEO Professor Phil Beales said the Axovia gene therapy platform gives hope to BBS patients worldwide. “In preclinical studies, our BBS1 novel gene therapy modified the underlying disease of BBS, including rescuing vision loss by halting retinal degeneration,” stated Professor Beales. Professor Beales is a renowned scientist and diagnostic leader in ciliopathies, having led research and patient care efforts out of University College London (UCL) Institute of Child Health for one of the most debilitating ciliopathic diseases in the world – BBS. See community resources here https://axoviatherapeutics.com/get-support-for-bbs/ “Our novel gene therapy utilises an adeno-associated virus (AAV9) to deliver a functional copy of the faulty BBS gene in key tissues,” he said. “Since AAV is not known to cause human disease and can be tightly controlled (it does not replicate like disease-carrying viruses), it has been the gene delivery method of choice for multiple therapies, including Luxturna for retinal disease.” Available clinical data on more than 3,000 people treated over more than 20 years indicate that AAV gene therapy is well-tolerated and efficacious. We are grateful to Fieldfisher LLP for their assistance on the transaction. For media inquiries, please contact: David JamesDigital Mantra GroupTeam@dmgpr.com About Axovia Therapeutics Axovia Therapeutics, an ALSA Ventures portfolio company, is developing the first novel gene therapies for ciliopathies and has a pipeline of products for these devastating diseases, including Bardet-Biedl Syndrome (BBS). Ciliopathies are a group of more than 40 rare inherited genetic diseases linked to more than 950 genes that impact the function of cilia. These microscopic finger-like organelles protrude from most cells in the body. The lead program AXV101 is planned to enter clinical trials in the next 18-24 months. AXV101 is an AAV9-based gene therapy targeting retinal dystrophy associated with BBS in patients carrying biallelic mutations in the BBS1 gene. It is designed to halt photoreceptor cell death and retinal degeneration. BBS is a rare disease affecting between 1 in 70,000 and 1 in 100,000 in Europe and North America. There is currently no cure. For further information, please visit https://axoviatherapeutics.com/ About ALSA Ventures ALSA Ventures is an early-stage investor in life sciences. The firm is building a portfolio of companies founded on innovative insights into disease treatment where current therapeutic options are either limited or non-existent. We work with a broad global network of preclinical and clinical experts to gain a deep understanding of translationally relevant disease biology and then leverage the team’s collective knowledge and operational experience to identify opportunities for drug intervention.Our focus is to improve patient outcomes by unlocking the full potential of clinical-stage assets. Our mission remains clear as we continue to grow our portfolio: to bring life-changing therapies to patients worldwide. For further information, please visit https://www.alsaventures.com/.