ASH

The first gene editing medicine and gene therapy for sickle cell disease were approved as the ASH meeting got under way, but novel oral drugs may be key to making treatment more accessible.

The recent ASH meeting began with US FDA approvals of the first genetic medicines for sickle cell disease, but doctors and biopharma companies still see a need for new oral therapies and other options. 

The drug maker provided updates at ASH on newer agents, including a GPRC5D-directed CAR-T therapy, a member of the CELMoD class and its entry into an increasingly crowded BCMA bispecific ring.

The company presented data for the menin inhibitor in KMT2Ar acute leukemias in an ASH late-breaker. The larger addressable population, including mNPM1 AML, could be a $2bn opportunity.

Autolus presented event-free survival rates and other updated results at ASH from its Phase Ib/II FELIX clinical trial of obe-cel, a CD19 CAR-T therapy recently submitted for US FDA approval.

Presentation of diffuse large B-cell lymphoma data at ASH showed low rates of higher-grade cytokine release syndrome and no cases of ICANS.