The US biotech’s DB-OTO has made headlines worldwide after a baby with profound genetic deafness experienced improved hearing to normal levels within 24 weeks after a single intracochlear injection.

The ranks of B7-H3-targeting antibody-drug conjugates under development in China have seen their first dropout, although elsewhere there are two FGFR2b-directed ADCs with first-in-class potential on the horizon. Meanwhile, there have been other discontinuations of KRAS G12C inhibitors and anti-BCMA CAR-T cell therapies in the country, while new players have surfaced in the areas of mRNA cancer vaccines and amyloid-beta-targeting antibodies for Alzheimer’s disease.

As sponsors explore development in autoimmune and infectious diseases, plus a range of hematologic and solid tumors, manufacturing processes can be centralized or decentralized, but Marks says the FDA is ‘neutral’ on that decision.

Early-stage companies pioneering new modalities discussed the future of cardiometabolic therapy and partnering with big pharma at the LSX conference in London.