Rocket Pharmaceuticals voluntarily withdrew the FDA submission for its Fanconi anemia gene therapy. The move follows a corporate restructuring over the summer that refocused the company on its cardiovascular programs.
Data on four of nine patients in a Phase I/II study presented at a conference in Spain met or exceeded the threshold agreed to with regulators for the company's Phase II trial of the therapy, RP-L102.
Zelis highlights how employers, payers, and consumers are integrating digital tools to manage healthcare costs.
New York biotech Abeona Therapeutics is taking a CRISPR/Cas9 approach to treating a rare anemia - and just raised $8.5 million in equity to bring it a little closer to clinic.