The FDA has granted approval for an Investigational New Drug application for Capsida Biotherapeutics' gene therapy, CAP-003, which targets Parkinson's disease linked to GBA mutations.

GBA mutations, present in up to 15% of Parkinson's disease cases, are a significant genetic risk factor for the condition.

CAP-003 specifically aims to treat Parkinson's associated with glucocerebrosidase (GBA) mutations, known as PD-GBA.

Swati Tole, Capsida's chief medical officer, emphasized the urgent need for therapies that target GCase, the protein related to the GBA gene, as current treatment options are inadequate.

The therapy utilizes an engineered capsid capable of crossing the blood-brain barrier, allowing for targeted delivery of genetic material while minimizing off-target effects.

This FDA approval enables Capsida to advance CAP-003 into clinical trials, marking their second IND clearance for a wholly owned clinical program.

Capsida plans to initiate a Phase I/II clinical trial for CAP-003, with the first patient expected to receive treatment in the latter half of 2025.

This decision reflects a broader trend of increasing regulatory support for innovative gene therapies within the biotech sector.

In related news, the biotech industry is witnessing significant consolidation, highlighted by BioNTech's recent acquisition of CureVac for $1.25 billion.

Additionally, SpliceBio has secured $135 million in Series B funding to advance its gene therapy SB-007 for Stargardt disease, a hereditary retinal disorder.

The funding will support ongoing Phase I/II studies and expand SpliceBio's pipeline, utilizing a proprietary protein splicing platform for precise gene delivery.

EQT Life Sciences and Sanofi Ventures co-led SpliceBio's funding round, which also saw participation from Roche Venture Fund and other investors.