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ALSA Ventures Launches Innovative Gene Therapy Company, Axovia Therapeutics, Focused on Ciliopathies Treatment

Wednesday, September 20, 2023

ALSA Ventures, a UK-based venture capital fund specializing in therapeutics, has made significant announcements regarding its recent acquisition of Axovia Therapeutics Inc. Alongside this acquisition, ALSA Ventures has initiated the launch of a new portfolio company known as Axovia Therapeutics Ltd.

Axovia Therapeutics is at the forefront of developing groundbreaking gene therapies for ciliopathies, a group of devastating diseases. These conditions include Bardet-Biedl Syndrome (BBS), and Axovia has a robust product pipeline aimed at addressing these disorders.

To expedite development, ALSA Ventures' investment team has devised an accelerated plan that aims to initiate clinical trials for their lead program, AXV101, within the next 18-24 months. The goal is to rapidly establish clinical proof of concept and seek regulatory approval.

AXV101 is an innovative gene therapy based on AAV9, designed to target retinal dystrophy associated with BBS in patients with biallelic mutations in the BBS1 gene. This therapy aims to halt the progressive retinal degeneration that typically begins in childhood and leads to blindness before the age of 20.

Revised epidemiological analyses indicate that BBS affects a significant portion of the population, estimated to be between 1 in 70,000 to 1 in 100,000 individuals in Europe and North America. Importantly, there is currently no available treatment for the retinal degeneration associated with BBS.

Alek Safarian, CEO of ALSA Ventures, expressed excitement about advancing AXV101 into clinical trials. He highlighted the importance of reaching well-characterized and motivated patient groups, particularly pediatric patients who require early intervention to prevent permanent vision damage.

Safarian also emphasized that ALSA Ventures was drawn to Axovia Therapeutics due to its impressive preclinical results, Rare Pediatric Disease Designation, and the proven AAV delivery mechanism. Furthermore, the company is well-positioned to pursue an FDA priority review voucher (PRV), a prestigious designation awarded to sponsors developing drugs for diseases, including rare pediatric conditions.

Axovia Therapeutics is the result of decades of research on ciliopathies conducted at University College London, led by co-founders Professor Phil Beales and Dr. Victor Hernandez.

Acting as CEO of Axovia, Professor Phil Beales expressed hope for BBS patients worldwide through the Axovia gene therapy platform. He highlighted that preclinical studies of their BBS1 gene therapy demonstrated the ability to modify the underlying disease, including halting retinal degeneration and rescuing lost vision.

Professor Beales, a renowned scientist and leader in ciliopathies, has played a pivotal role in advancing research and patient care efforts at the University College London (UCL) Institute of Child Health, particularly for BBS.

The gene therapy developed by Axovia utilizes an adeno-associated virus (AAV9) to deliver a functional copy of the faulty BBS gene in critical tissues. AAV is a preferred gene delivery method due to its safety record; it does not cause human disease and can be tightly controlled, unlike disease-carrying viruses. Notably, AAV gene therapy has proven to be well-tolerated and effective, with available clinical data spanning over 20 years and involving more than 3,000 treated individuals.

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