LONDON, April 28, 2025 (GLOBE NEWSWIRE) -- Axovia Therapeutics Ltd., a biotechnology company developing therapies to address the genetic causes of blindness and obesity, today announced that Co-Founder and Chief Scientific Officer, Dr. Victor Hernandez, will present new preclinical data in three poster presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which is being held from May 13-17, 2025, at the New Orleans Ernest N. Morial Convention Center in New Orleans, LA.
Two poster presentations include preclinical data that support the dosing regiment and study protocol for the subretinal clinical study for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, which the company expects to initiate in mid-2025. A third poster unveils the first preclinical proof of concept data for novelly designed, AXV-201, to treat individuals with severe obesity and very high BMIs resulting from a MC4R mutation.
Presentation details:
Title: A new dosing regimen to treat Bardet-Biedl Syndrome 1 (BBS1) retinal degeneration with AXV-101 (AAV9-BBS1) improves histological and functional photoreceptor survival in Bbs1 M390R mice. Finding the right balance between bleb number, dose per bleb and total dose per eye
- Abstract ID/Number: AMA283
- Type: Poster Session
- Location: Poster Hall Hall I2
- Date/Time: Tuesday, May 13, 2025 from 6:00 PM - 7:30 PM CDT
Title: Development of a ddPCR Assay to Quantify AXV-101 Levels for a Mouse Biodistribution Study
- Abstract ID/Number: AMA1165
- Type: Poster Session
- Location: Poster Hall Hall I2
- Date/Time: Wednesday, May 14, 2025 from 5:30 PM - 7:00 PM CDT
Title: A codon-optimised human sequence of human MC4R regulates cAMP levels and normalise weight in Mc4r mutant obese males and females when delivered with as a self-complementary AAV9 (scAAV9) with an intracerebroventricular delivery
- Abstract ID/Number: AMA284
- Type: Poster Session
- Location: Poster Hall Hall I2
- Date/Time: Thursday, May 15, 2025 from 5:30-7:00 p.m. CDT
About Bardet-Biedl Syndrome (BBS)
Bardet-Biedl Syndrome (BBS) is an autosomal recessive disorder associated with primary cilia dysfunction, presenting with retinal degeneration and morbid obesity, amongst other clinical features. There is no curative treatment for BBS. BBS affects between one in 70,000 - one in 100,000 in Europe and North America, with up to 10 times that prevalence in certain populations in the Middle East. BBS1 is the most commonly mutated gene found in BBS, with the missense BBS1 M390R mutation being the most common allele.
About Melanocortin 4 Receptor (MC4R) Mutations
Melanocortin 4 receptor (MC4R) mutations are the most common cause of human monogenic obesity. Individuals with MC4R mutations have hyperphagia, severe obesity and hyperinsulinemia. Impact of the different type of MC4R mutation depends on how they reduce MC4R expression and the impact they have on the production of cyclic AMP (cAMP). Mutations in heterozygosity, with partial MC4R functionality are the most frequent, but homozygous mutations and double heterozygotes account for 25% of MC4R mutations. There are approximately 50,000 patients with complete MC4R loss of function (LoF) and BMI>40 in the United States, European Union and Middle East.
About Axovia Therapeutics Ltd.
Axovia Therapeutics is leading the development of therapies that address the genetic causes of blindness and obesity syndromes that are driven by cilia dysfunction. Ciliopathies are a group of more than 40 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. The company plans to initiate a clinical study to treat retinal degeneration for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, in mid-2025, based on robust preclinical efficacy and toxicological data with established scaled GMP manufacturing and patient registries. AXV-101 has achieved U.S. Food and Drug Administration Orphan Drug Designation and Rare Pediatric Disease Designation. The company is developing its second program, AXV-201, for genetic obesity caused by MC4R mutations. Axovia is backed by ALSA Ventures and was formed following decades of work on ciliopathies at University College London by co-founders Professor Phil Beales and Dr. Victor Hernandez. For further information, please visit https://axoviatherapeutics.com.
Contacts:
Professor Phil Beales
Chief Executive Officer
investors@axovia.com
