Sangamo Therapeutics Inc. is adding a much-needed $18 million up-front payment in a neurology-focused deal with Eli Lilly and Co. that could bring up to an additional $1.4 billion. In return, Lilly gets access to Sangamo’s neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has shown early promise in penetrating the blood-brain barrier penetration, for one initial target with the right to add up to four more.
Sangamo Therapeutics Inc.’s gene therapy for Fabry disease, isaralgagene civaparvovec (ST-920), is continuing to show promising efficacy and safety in the phase I/II study, Staar, but further ahead, the company needs to either attract a partner or secure financing to move to a registrational trial.
Sangamo Therapeutics Inc. rolled out pleasing preliminary data from the first four patients treated in the phase I/II study known as Staar, evaluating isaralgagene civaparvovec, or ST-920, a gene therapy for Fabry disease. Results as of the Sept. 17, 2021, cutoff date from the four patients in the first two dose cohorts showed that the drug was generally well-tolerated, and all four patients exhibited above normal alpha-galactosidase A activity.
