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news.US-based gene therapy company Spark Therapeutics has started enrollment in a Phase I/II clinical trial of its product candidate, SPK-CHM, to treat patients with choroideremia (CHM), an X-linked inherited retinal dystrophy.
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The open-label, dose-escalating Phase I/II trial is designed to evaluate the safety and preliminary efficacy of sub-retinal administration of SPK-CHM.
Spark co-founder and chief executive officer Jeffrey Marrazzo said: "Choroideremia is a rare, blinding condition which affects males most severely at middle age, a critical time personally and professionally."
Currently, the company is planning to enroll up to ten patients afflicted with the CHM genetic mutation.
Apart from evaluating safety, the trial will help define the dose required to achieve stable or improved visual function and identify suitable endpoints for subsequent clinical trials.
With SPK-CHM, the company is leveraging the experience and technology used in the development of its lead Phase III program, SPK-RPE65, including the same vector, target cells and route of administration, as well as the same manufacturing process.
In preclinical trials, SPK-CHM showed the ability to restore REP-1 protein production, intracellular trafficking and retinal structure.
These trials were conducted in collaboration with Jean Bennett, the F.M. Kirby Professor of Ophthalmology in the Perelman School of Medicine at the University of Pennsylvania (Penn), and the Center for Retinal and Ocular Therapy (CAROT) at Penn.
The Phase I/II trial will be conducted at The Children’s Hospital of Philadelphia (CHOP) and Penn, leveraging the same clinical study teams that carried out the Phase I and Phase IIII clinical trials of SPK-RPE65.