Cell therapy weekly: cell therapy for vision loss progresses to clinical trial
This week: Sanaregen Vision Therapeutics (TN, USA) will progress its gene therapy for Familial Drusen to clinical trial following approval from the US Food and Drug Administration (FDA; MD, USA), the Progeria Research Foundation (PRF; MA, USA) and Forge Biologics (OH, USA) have entered into a manufacturing agreement to support the development of a gene therapy for Hutchinson-Gilford Progeria Syndrome (Progeria), and Cure Rare Disease (CRD; CT, USA) has partnered with the LGMD2L Foundation (IL, USA) to develop a gene replacement therapy for the rare genetic disorder Anoctamin 5 (ANO5)-related disease.
The news highlights:
- Cell therapy for vision loss progresses to clinical trial
- Progeria gene therapy manufacturing agreement
- Partnership drives development of rare disease gene therapy
Cell therapy for vision loss progresses to clinical trial
The FDA has granted Sanaregen Vision Therapeutics’ clearance to commence a Phase I/II clinical trial for its investigational cell therapy for Familial Drusen, an inherited form of macular degeneration. The trial will assess safety and effectiveness of SVT-001 to improve retinal function and restore vision in individuals with Familial Drusen.
“This is a pivotal moment not only for our company, but for the families that have endured generations of early-onset blindness,” said Anthony Oliva, Chief Scientific Officer and Co-Founder. “We believe SVT-001 provides true hope to prevent the almost inevitable vision loss caused by this devastating condition that has largely been overlooked.”
Progeria gene therapy manufacturing agreement
PRF and Forge Biologics have entered into a manufacturing agreement to support the development and manufacturing of PRF’s investigational gene therapy for Progeria, SamPro-2. The in vivo gene-editing therapy utilizes an AAV9 capsid to deliver CRISPR-based gene editing technology to correct the single DNA base mutation in the lamin A gene that causes Progeria.
“The era of Progeria gene therapy has arrived. Our hope is that SamPro-2 will give children and young adults with Progeria the longer, healthier lives they deserve,” said Leslie Gordon, Co-Founder and Medical Director of PRF and the mother of Sam Berns who had Progeria. “We are extremely grateful to be working with Forge Biologics, whose manufacturing expertise is essential to move this work from the laboratory towards clinical trials.”
Under the agreement, Forge Biologics will provide process development and manufacturing expertise to support the advancement of SamPro-2 towards an Investigational New Drug application.
Partnership drives development of rare disease gene therapy
CRD has partnered with the LGMD2L Foundation to develop a gene replacement therapy for ANO5-related disease, a rare genetic disorder. The collaboration is supported by US$7.65 million in funding from the LGMD2L Foundation, which will drive the therapy’s development from initial design to clinical trial readiness.
The multi-year program will focus on key milestones, including therapeutic design, preclinical studies, manufacturing and a first-in-human clinical trial. This partnership also highlights CRD’s broader pipeline, which includes gene therapies for other rare neuromuscular diseases like LGMD2i/R9 and LGMD2g/R7, utilizing next-generation AAV capsids designed for improved safety.
“Gene therapy is the most promising therapeutic avenue for this devastating disease, yet no private-sector programs are currently advancing this approach,” said Hal Tily, VP of Research for the LGMD2L Foundation. “This partnership marks a pivotal moment for patient-led organizations in shaping their own futures. CRD’s track record navigating the complexities of therapeutic development gives us strong confidence that this investment can change the lives of individuals and families affected by LGMD2L.”