In an historic decision, the U.S. Food and Drug Administration has approved Itvisma, an adeno-associated virus vector-based gene therapy for spinal muscular atrophy. By extending eligibility to adults and children two years of age and older, this new treatment offers fresh hope for families coping with the disease.

uniQure N.V. (QURE) is gaining bullish attention for its pioneering AMT-130 gene therapy, which some observers view as a groundbreaking approach to Huntington’s disease. With shares trading at $63.65 and a forward P/E of 27.93, investors see promise in the biotech’s innovative push toward a first-of-its-kind treatment.

In a landmark move, the Chan Zuckerberg Initiative and the Innovative Genomics Institute have announced a new center dedicated to bringing personalized CRISPR cures to children with rare genetic diseases.