Pipeline
We may be science-led, but we’re people-focused
Pipeline
The company is positioned to initiate clinical studies for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, in mid-2025 based on robust preclinical efficacy and toxicological data, established scaled manufacturing and strong patient community engagement and a leading comprehensive patient registry. The initial subretinal study is designed to halt photoreceptor cell death and prevent further retinal degeneration and the CNS delivery program, which will begin in 2026, will seek to address hyperphagia and obesity. 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.
| Disease | Program | 2025 | 2026 | 2027 | 2028 | ||
|---|---|---|---|---|---|---|---|
| Ciliopathies | |||||||
| Disease: BBS1 | Program: AXV101 (SR) | Stage: Pre-Clinical | |||||
| Disease: BBS1 | Program: AXV102 (ICV) | Stage: Pre-Clinical | |||||
| Disease: MC4R | Program: AXV201 (ICV) | Stage: Discovery | |||||
- Discovery
- Pre-Clinical
- First in Human
AVX-101
What is a clinical trial?
A clinical trial is a research study conducted by doctors, with human volunteers (participants), to evaluate the safety, effectiveness and potential side effects of new medical treatments, drugs, devices, or interventions.
Clinical trials are carefully designed and follow strict protocols to answer specific health questions. They are a key step in the development of new therapies and are essential for drug approvals by regulatory agencies such as the Medicines and Healthcare products Regulatory Agency (MHRA), European Medicines Agency (EMA) or Food and Drug Administration (FDA).
What should I know about participating in a clinical trial?
Clinical trials study potential new treatments and are voluntary. Every trial has potential risks (such as side effects) and possible benefits (such as access to new treatments).
If you’re considering participating in a clinical trial, it’s wise to discuss with your healthcare provider and reach out to patient advocacy organizations for additional support and information.
What is the purpose of the clinical trial?
This is a gene therapy study to assess the safety, tolerability and effectiveness of AXV-101 being injected into one eye in participants (4-17 years) with BBS1 bi-allelic (two) mutations and retinal degeneration. The treated eye will be compared with the eye not injected with AXV-101.
How does AXV-101 work as a treatment?
The injection contains a “working” copy of the faulty BBS1 gene, packaged inside a viral shell (a common method in gene therapy). The new gene enters the retina cells, which “read” the healthy gene and start making the missing protein again. By restoring this protein, the therapy helps keep the retinal cells functioning and aims to slow or even stop further vision loss.
How can I learn more about the clinical study and eligibility to participate?
To learn more about the AXIS trial, sponsored by Axovia Therapeutics, and eligibility criteria for participation, please visit the official clinical study page at ISRCTN or clinicaltrials.gov.
Where is this trial being conducted?
This is a prospective, open-label clinical trial where all participants know they are receiving the investigational gene therapy product and will be followed for safety and efficacy for 5 years after dosing. Participants will need to come to the clinical trial site 19 times for assessments and follow-ups during the trial.
What is the route of administration into the eye?
The investigational gene therapy is given as a sub-retinal injection, meaning the therapy is injected underneath the retina during a surgical procedure.
Who can I contact for more information about the trial?
Further information for the trial is available at ISRCTN or clinicaltrials.gov
If you would like to register your interest in the AXIS clinical trial please email moorfields.resadmin@nhs.net with your details and cite the reference study number: MOOM1022 /IRAS ID: 1011952