About
We are developing new therapies to treat diseases caused by cilia dysfunction
About
Axovia Therapeutics is leading the development of therapies that address the genetic forms of blindness and obesity associated with ciliopathies. Ciliopathies are a group of more than 55 rare inherited genetic diseases linked to more than 950 genes that impact the function of cilia which are critical for protein transport and cellular signaling.
FOCUS ON CILIOPATHIES
CILIOPATHIES SUCH AS BBS HAVE NO DISEASE-MODIFYING THERAPIES AVAILABLE
Ciliopathies are a group of more than 55 rare inherited genetic diseases linked to more than 950 genes that impact the function of cilia, microscopic finger-like appendages that sit atop most cells in the body.
Those with ciliopathic symptoms generally experience a “rare disease odyssey” where these patients report having waited 5-30 years for the correct diagnosis, with the initial diagnosis being incorrect in 40% of cases.
Our lead development program focus is on Bardet-Biedl Syndrome (BBS) which is known to cause at least 16 severe or life-threatening medical conditions including visual impairment that leads to early blindness, severe life-limiting obesity, learning difficulties, life-threatening kidney abnormalities, urogenital dysfunction, complex endocrine dysfunction and post-axial polydactyly (additional digits outside little fingers or toes).
OUR COMMITMENT TO CILIOPATHIES
After decades of helping people with ciliopathies to manage medical conditions without dedicated treatments, Prof. Philip Beales and Dr. Victor Hernandez co-founded Axovia to develop treatments to address the disease burden for this group of disorders with a focus on preventing blindness and reversing obesity.
Robust preclinical data suggest that our BBS1 novel gene therapy can modify the underlying disease of BBS, including rescuing vision loss by halting retinal degeneration and correcting the genetic cause of hyperphagia and morbid obesity, hallmarks of BBS mutations. The potential to preserve vision and reverse obesity in patients with BBS1 would have profound and dramatic impact on the health and well-being of this community and we expect to begin an initial clinical trial in mid-2025.
Our second program targets the most common genetic cause of obesity. We expect to advance this program into clinical testing in 2026.
OUR APPROACH
AAV PLATFORM
Our novel gene therapy utilizes an adeno-associated virus (AAV9) to deliver a functional copy of the faulty BBS gene into key tissues.
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 a number of therapies including Luxturna for retinal disease and Zolgensma for spinal muscular atrophy.
Available clinical data, covering more than 3,000 people treated over more than 20 years, indicate that AAV gene therapy is a well-tolerated and efficacious form of therapy.
Our Team
Phil Beales, MD FRCP FMedSci
Chief Executive Officer & Co-Founder
Professor Phil Beales is the Chief Executive Officer at Axovia. In 2020, he co-founded the company with Dr. Victor Hernandez as a spin-off from the University College London. Prof Beales is a renowned scientist and diagnostic leader in ciliopathies, having led the most comprehensive research and care effort for one of the most debilitating ciliopathic diseases in the world - Bardet-Biedl syndrome (BBS). Over the past 25 years, some of Prof Beales’ most notable achievements include establishing the diagnostic criteria for BBS, co-identifying the first BBS gene, demonstrating the role of cilia in BBS and other ciliopathies, and playing an instrumental role in advancing care for patients suffering from BBS. He was instrumental in opening up the first specialized clinic for BBS patients in the UK, funded by the NHS.
He gained his undergraduate degrees in genetics and medicine from University College London and postgraduate degree from Kings College London. He is an elected Fellow of the Academy of Medical Science.
Victor Hernandez, Ph.D.
Chief Scientific Officer & Co-Founder
Dr. Victor Hernandez serves as a Axovia’s Chief Scientific Officer. In 2020, he co-founded the company with Prof Phil Beales as a spin-off from the University College London. In 2008, he joined the Prof Phil Beales team at the Institute of Child Health, University College London, to investigate the cellular mechanisms of genes linked to ciliopathies and in 2012, started leading the development of gene therapies solutions for Bardet-Biedl syndrome (BBS). In 2019 he joined Brunel University London as a Senior Lecturer in Biomedical Sciences. Victor completed his Ph.D. at the Genetics Department of the Universitat de Barcelona.
Lorna Collings
Chief Financial Officer
Lorna Collings has more than twenty five years of experience in private equity and venture capital, with roles at EQT, Hermes GPE, Coller Capital, Ariadne Capital and BancBoston Capital. She is currently the Chief Financial Officer of the ALSA Ventures Group. She holds a BA in Applied Psychology from the University of Sussex and is a Chartered Management Accountant (GCMA).
Bruce Goldsmith, Ph.D.
Investor Relations
Dr. Bruce Goldsmith is an Operating Partner with ALSA Ventures and has a strong background in Pharma and Biotech leadership over the past 25 years. He held progressively senior roles at Pharma and Biotech companies, including Johnson & Johnson, Allos Therapeutics and Lycera Corp. Following this experience, he joined Deerfield Healthcare as a Venture Partner and led a new company creation as CEO. He then joined Passage Bio as CEO leading an IPO and advancing four programs through IND. Throughout his career Bruce has led business development deals, strategy and operations. Bruce holds a Ph.D. in Biology from the University of Pennsylvania and an MBA from Columbia Business School.
Jo Broadhead, Ph.D.
CMC Lead (Consultant)
Dr. Jo Broadhead is a CMC and Programme Management Consultant, primarily supporting AAV gene therapy start-up companies as they progress towards and through clinical trials. Prior to forming BioCMC Consulting at the beginning of 2022, Jo spent over 5 years at Freeline Therapeutics, where she led the CMC projects and CMC regulatory teams, including supporting multiple clinical trial submissions in Europe and the US for the company’s AAV gene therapy portfolio. Prior to joining Freeline, Jo held a 4 year interim role supporting late phase product development for an MVA viral vaccine developer. Earlier in her career, she spent over a decade at AstraZeneca in various parenteral product development and manufacturing roles. Jo also spent a short period of time as a lecturer at DeMontfort University and was actively involved in the development of their distance learning Quality by Design course. Jo has been an active member of various industry groups throughout her career including the Academy of Pharmaceutical Scientists and the American Society of Cell and Gene Therapy. Jo has a degree in Pharmacy from the University of Bath and a Ph.D. in Pharmaceutical Sciences from the University of Rhode Island.
Elizabeth Forsythe, MBBS, BMedSci, PhD
Medical Director (Consultant)
Dr. Elizabeth Forsythe is a Clinical Geneticist Consultant with a particular focus on clinical trials and new therapies for rare diseases. She has expertise in ciliopathies and rare obesity syndromes and is one of the lead clinicians for the UK National Bardet-Biedl Syndrome Clinics. Dr. Forsythe has successfully led clinical trials into an investigational medicinal product for a rare genetic disorder, provided expertise for the National Institute of Clinical Excellence leading to approval, and now runs a treatment clinic. Dr. Forsythe's clinical expertise is complemented by her active involvement in research and patient advocacy. She serves on the Board of Trustees for the Ciliopathy Alliance, a UK charity dedicated to supporting individuals affected by ciliopathies. She is also a member of the Bardet-Biedl syndrome (BBS) Scientific Advisory Board, contributing her expertise to guide research and clinical practices related to BBS. She obtained her PH.D. from University College London.
Charmaine O’Neill
Vice President Clinical Operations
Charmaine brings over 30 years of diverse experience within Research and Development and currently supports Axovia Therapeutics and Vantage Biosciences. She was most recently at Gyroscope Therapeutics, a Novartis company. She has held key senior roles across the pharmaceutical and biotech industry, such as GSK, Aimmune Therapeutics, part of Nestle Health Sciences, DSPE, PPD, Syneos and hVivo. With a rich background in various therapeutic areas, including ophthalmology, virology, diabetes, rare diseases, food allergy and oncology, she has successfully managed Phase I-III study programmes through to registration. Charmaine holds BSc (Hons)’s in Pharmacology and Psychology.
Narges Amini, Ph.D.
Programme Director
Dr. Narges Amini possesses a diverse background across academia, clinical research, and industry spanning Europe and the Middle East. As a Programme Director, she contributed to the establishment of Axovia Therapeutics. Previously, she served as an Innovation and Commercialisation Manager at QBRI (Qatar). Before that, while at UCL, she held roles in the Business & Innovation Group and provided founding leadership for the P4 Precision Medicine Accelerator. Narges also brings valuable expertise from her tenure at Imperial Innovations, GlaxoSmithKline, and UCL Cancer Trial Centre. She holds a Ph.D. from Imperial College London, complimented by an MSc and BSc in Genetics.