At the current pace of innovation in the U.S. rare disease space, developing and approving therapies for just half of the 10,000-plus known rare diseases would take more than 160 years, Bradley Campbell, president and CEO of Amicus Therapeutics Inc., recently told the Senate Committee on Aging.

Sanaregen Vision Therapeutics Inc. has received FDA clearance to conduct a phase I/II trial of SVT-001, an investigational cell therapy for individuals with familial drusen.

CAR T cells have been groundbreaking for the treatment of B-cell cancers. But 8 years after Kymriah (tisagenlecleucel, Novartis AG) became the first CAR T-cell therapy to be approved, there are no CAR Ts approved for solid tumors.