Sangamo anounces data from neurotropic AAV capsid showing BBB penetration

Sangamo Therapeutics announced preclinical data from its proprietary adeno-associated virus AAV capsid variant, STAC-BBB, that demonstrated robust penetration of the blood-brain barrier, BBB, and strong transgene expression throughout the central nervous system, CNS, of NHPs when administered intravenously at clinically-relevant doses, outperforming results obtained by Sangamo for other known neurotropic capsid variants evaluated in the study. Importantly, potent repression of target genes was observed in brain cells expressing the zinc finger cargo, indicating that STAC-BBB could enable development of genomic medicines to potentially treat a wide range of neurological diseases. “The advancement of neurological medicines has long been limited by the inability to achieve widespread CNS delivery, an essential attribute for an effective treatment for devastating neurological disorders. We are extremely encouraged that STAC-BBB, a potentially game-changing capsid variant engineered using our SIFTER capsid platform, demonstrated results that outperformed other known neurotropic capsid variants, achieving widespread brain delivery and transgene expression, desired de-targeting of liver and other peripheral tissues and a favorable safety profile,” said Sandy Macrae, Chief Executive Officer of Sangamo. Sangamo intends to use the novel STAC-BBB capsid in its wholly owned prion disease and tauopathy programs. In addition, STAC-BBB could potentially unlock multiple neurology epigenetic regulation programs that were paused by Sangamo pending the identification of a suitable capsid, including programs previously in development under Sangamo’s former collaboration agreements with Biogen and Novartis. Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators