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This week: The first patient has been dosed in a clinical trial investigating an induced pluripotent stem cell (iPSC)-derived regenerative cell therapy for retinitis pigmentosa (RP), the US Food and Drug Administration (FDA; MD, USA) has allowed expanded enrollment in a trial investigating a treatment for Duchenne muscular dystrophy derived from umbilical cord mesenchymal stem cells, and a gene therapy for pediatric deafness has reached clinical trial
The news highlights:
- First patient dosed with iPSC therapy for retinitis pigmentosa
- Expanded enrollment for Duchenne muscular dystrophy stem cell therapy
- Gene therapy for pediatric deafness reaches clinical trial
First patient dosed with iPSC therapy for retinitis pigmentosa
The first patient has been dosed in Sumitomo Pharma America’s (MA, USA) Phase I/IIa study evaluating DSP-3077 for the treatment of non-syndromic RP. DSP-3077 is an investigative allogeneic induced pluripotent stem cell (iPSC)-derived regenerative cell therapy that is administered
DSP-3077 utilizes a three-dimensional neural tissue cultivation approach called the SFEBq method, which was created by Yoshiki Sasai and his team at RIKEN (Tokyo, Japan). This technique enables pluripotent stem cells to develop into organized neural structures and organoids through self-directed cellular organization
“We are encouraged by the potential that iPSCs may hold for treating degenerative, debilitating conditions like RP that currently have few therapeutic options, and this first patient treated is a notable milestone for our company,” said Tsutomu Nakagawa, President and CEO of SMPA. “Being able to provide this investigational treatment to our first study participant is not only a great honor, but a very important milestone in the development of DSP-3077, one that will aid in better understanding how it and future iPSC therapies could help improve the lives of RP patients and their families.”
Expanded enrollment for Duchenne muscular dystrophy stem cell therapy
Signature Biologics (TX, USA), a cell therapy company focused on clinical research for Duchenne muscular dystrophy, has received FDA approval to increase participant enrollment in its clinical trial examining an experimental treatment derived from umbilical cord mesenchymal stem cells
The trial operates under an Investigational New Drug application managed by a contracted clinical research organization acting as sponsor, while Signature Biologics produces the experimental therapy and provides financial support
Five participants have been enrolled in the initial group and the FDA has authorized enrollment of up to 30 more participants. Recruitment will continue once an additional clinical location is established and operational
Gene therapy for pediatric deafness reaches clinical trial
Skylark Bio (MA, USA), a biotechnology company specializing in genetic treatments for hearing loss and single-gene disorders, has announced the administration of its first dose in the SONIX Phase I/II trial. The study is evaluating SKY-GJB2, an experimental gene therapy targeting the inner ear for hearing loss caused by GJB2 gene mutations. The company is developing a specialized genetic medicine platform for single-gene diseases that currently lack approved treatments, with a focus on precise localized delivery and targeted cellular expression.
“Dosing the first patient in SONIX brings us one step closer to a future where children born with genetic hearing loss have new treatment options that could meaningfully change the trajectory of their lives. SONIX is designed to generate preliminary data by year-end 2026 with additional readouts anticipated in 2027. The recent approval of a targeted inner-ear AAV-delivered therapy provides important validation for the field and further reinforces our confidence in this approach,” said Jodi Cook, CEO of Skylark Bio. “We are honored by the trust of participating families and grateful to the investigators and site teams supporting this study.”
