A genetic disorder that leads to bleeding episodes and life-threatening infections has a new FDA approved treatment, a regulatory decision that marks two notable firsts — the first gene ...
Regeneron Pharmaceuticals is committing $150 million to begin an alliance on a Tessera Therapeutics gene-editing medicine nearing the clinic as a potential treatment for a rare disease ...
Light chain amyloidosis is not cancer, but cancer drugs are this rare disease’s standard of care. The patient need for new treatment options has sparked R&D efforts at biotech and big ...
