Spur Therapeutics Highlights Promising AAV Gene Therapy Data for Gaucher, Parkinson’s, and AMN

LONDON, May 15, 2025 (GLOBE NEWSWIRE) –Spur Therapeutics announced positive new data showcasing the potential of its AAV gene therapy programs targeting Gaucher disease, GBA1 Parkinson’s disease, and adrenomyeloneuropathy (AMN).

Chief Medical Officer Dr. Pamela Foulds emphasized their tailored AAV gene therapy approach. The lead AAV program, FLT201 for Gaucher disease, demonstrated strong safety and efficacy for nearly two years after a single dose, utilizing an AAV vector to deliver a more stable enzyme. As they prepare for a Phase 3 trial for this AAV therapy, these results are encouraging. Promising preclinical findings were also presented for their Parkinson’s AAV gene therapy, SPR301, which uses the same engineered enzyme optimized for brain expression.

Durable Benefit with AAV Gene Therapy for Gaucher Disease

Updated Phase 1/2 data from the GALILEO-1 trial of FLT201, an AAV gene therapy for Gaucher type 1, showed sustained positive outcomes in four patients who stopped prior therapies for up to 21 months after a single low dose of the AAV. This AAV treatment led to robust, sustained reductions in lyso-Gb1, stable hematological parameters, and preclinical data in NHPs showed long-term GCase activity following AAV administration. Transient antibodies to the delivered enzyme did not impact clinical benefit in one patient.

Encouraging Preclinical AAV Gene Therapy for Parkinson’s

Preclinical data for SPR301, an AAV gene therapy leveraging the same stable enzyme as FLT201 but optimized for brain delivery, showed superior enzyme exposure and distribution in key brain regions of mice with Parkinson’s-related deficiencies, compared to wildtype AAV gene therapy. This AAV approach also demonstrated better substrate reduction and α-synuclein reduction in lab studies.

Tolerable AAV Gene Therapy for AMN

A safety update from the Phase 1/2 PROPEL trial of SBT101, an AAV gene therapy for AMN, indicated it continues to be generally well-tolerated in eight patients over a significant follow-up period, with mostly non-serious adverse events reported.

In conclusion, Spur Therapeutics highlighted the potential of their AAV gene therapy approaches to significantly improve outcomes for these serious genetic diseases, with compelling clinical data in Gaucher disease and encouraging preclinical results in Parkinson’s disease. The tolerability of their AAV therapy for AMN also supports its continued development.