Viralgen and Axovia Therapeutics enter partnership, target retinal dystrophy in Bardet-Biedl Syndrome

News
Article

The lead gene therapy candidate, AXV-101, is expected to enter clinical development in mid-2025

An illustration of a genetic DNA helix with a red target on it. Image credit: ©Елена Бутусова – stock.adobe.com

Viralgen and Axovia are developing an AAV9-based gene therapy for retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS). Image credit: ©Елена Бутусова – stock.adobe.com

Viralgen and Axovia Therapeutics are partnering to advance the development and manufacture of an AAV9-based investigational gene therapy aimed at treating retinal dystrophy in patients with Bardet-Biedl Syndrome (BBS). The collaboration will give patients access to a dynamic investigational treatment with the potential to stop photoreceptor cell death and retinal degeneration, as well as halt vision loss.

According to a press release, the two companies will work together by using the codon-optimised BBS1 AAV9 vector to minimise the vision loss caused by the genetic defects in the BBS1 gene. The therapy will also be manufactured at Viralgen’s facility, capitalising on the company’s expertise in AAV and using the Pro10 cell line and manufacturing platform.

“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 [Contract Development and Manufacturing Organisation],” said Victor Hernandez, co-founder and chief scientific officer. “Our lead programme, AXV-101, which is being developed to address retinal dystrophy associated with Bardet-Biedl Syndrome (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.”

“We are delighted to partner with Axovia and bring in our AAV manufacturing expertise. We are committed to supplying quality vectors and timely supply with the goal of providing disease-modifying treatment for patients affected by BBS1-related retinal dystrophy,” Jimmy Vanhove, CEO of Viralgen, said.

Reference

Viralgen and Axovia Partner to Manufacture an Investigational Gene Therapy for Retinal Dystrophy in Bardet-Biedl Syndrome (BBS) patients. Viralgen. Published January 22, 2025. Accessed January 27, 2025. https://viralgen.com/news/viralgen-and-axovia-partner-to-manufacture-an-investigational-gene-therapy-for-retinal-dystrophy-in-bardet-biedl-syndrome-bbs-patients

Newsletter

Join ophthalmologists across Europe—sign up for exclusive updates and innovations in surgical techniques and clinical care.

Recent Videos
Viha Vig, MBChB graduate student at the University of Auckland, New Zealand, discusses her poster presentation on the relationship between mitochondiral disease, Alzheimer disease, and other types of dementia.
Alfredo Sadun, MD, PhD, chief of Ophthalmology at the Doheny Eye Institute, University of California Los Angeles, shared exciting new research with the Eye Care Network during the Association for Research in Vision and Ophthalmology (ARVO) meeting on the subject of Leber hereditary optic neuropathy (LHON).
At this year's Association for Research in Vision and Ophthalmology (ARVO) meeting in Salt Lake City, Utah, Nitish Mehta, MD, shared highlights from his research documenting real-world results of aflibercept 8 mg for patients with diabetic macular oedema.
ARVO 2025: Anat Loewenstein, MD, shares data from herself and her colleagues on meeting needs of patients with diabetic retinopathy
At the American Society of Cataract and Refractive Surgeons annual meeting, Sheng Lim, MD, FRCOphth discusses the benefit of endoscopic cyclophotocoagulation for patients with primary open angle glaucoma and cataracts in the CONCEPT study
A photo of Seville, Spain, with the Congress on Controversies in Ophthalmology logo superimposed on it. Image credit: ©francovolpato – stock.adobe.com; logo courtesy COPHy
Anat Loewenstein, MD, Professor and Director, Department of Ophthalmology, Tel Aviv Medical Center, discusses the Congress on Controversies in Ophthalmology (COPHy)
Anat Loewenstein, MD, speaks about the 22nd Annual Angiogenesis, Exudation, and Degeneration Meeting in February 2025 and shares her global forecast for AI-driven home OCT
Sarah M. Thomasy, DVM, PhD, DACVO, a veterinary ophthalmologist at UC Davis, talks about how her research at the Glaucoma 360 symposium
I. Paul Singh, MD, an anterior segment and glaucoma specialist, discusses the Glaucoma 360 conference, where he participated in a panel discussion on the use of artificial intelligence (AI) in glaucoma care.
© 2025 MJH Life Sciences

All rights reserved.