The first patient has been dosed in a phase 1/2 trial of GB221, a next-generation gene therapy for spinal muscular atrophy type 1. The FDA has granted GB221 Rare Pediatric Disease designation, potentially qualifying Gemma Biotherapeutics for a Priority Review Voucher.
The first patient has been dosed in a phase 1/2 trial of GB221, an SMA gene therapy delivered via intra-cisterna magna injection. The US Food and Drug Administration (FDA) has granted the therapy Rare Pediatric Disease designation. FDA approval of GB221 could qualify Gemma Biotherapeutics for a Priority Review Voucher. SMA is a rare neurodegenerative disorder involving progressive muscle weakness and loss of movement. Until 2016, management focused on supportive care and overall quality of life. The identification of SMN1 abnormalities as the underlying cause of SMA enabled development of targeted treatments, 4 of which have obtained FDA approval. The gene therapy utilizes a proprietary serotype hu68 adeno-associated virus vector. It carries a codon-optimized SMN1 transgene, which is contained within an expression cassette modified to limit overexpression and sensory neurotoxicity. CHARISMA is the first clinical trial to evaluate an SMA type 1 gene therapy delivered via the cisterna magna route. GB221 is the first SMA type 1 gene therapy delivered via intra-cisterna magna injection to reach the clinical trial stage. Close more info about First Patient Dosed in Clinical Trial of GB221, Next-Generation Gene Therapy for SMA Type 1
Original title: “First Patient Dosed in Clinical Trial of GB221, Next-Generation Gene Therapy for SMA Type 1”