Beam base editing therapy gets ‘proof of concept’ in rare lung disease

While initial study results suggest Beam's technology can correct alpha-1 antitrypsin deficiency's genetic roots, shares fell by double digits.

MeiraGTx spins Parkinson’s, obesity gene therapies into AI startup

The joint venture with generative AI firm Hologen hands MeiraGTx $200 million up front as well as other financial perks — a “transformative” deal, according to the company’s CEO.

Accelerate CRISPR-Cas characterization: From design to innovation in 8 days

Ono pays $280M to license Ionis rare disease drug

Ionis’ drug, called sapablursen, is currently being tested in a Phase 2 trial in people with polycythemia vera.

Roche launches new Boston center; NIH centralizes peer review

The Swiss pharma will base cardiometabolic drug research at the new Allston site. Elsewhere, AskBio, Bayer’s gene therapy subsidiary, advanced an experimental treatment for limb-girdle muscular dystrophy. 

Once vaunted as a cure, gene therapy has hit hard times

For Patients Needing Transplants, Hope Arrives on Tiny Hooves

CBER Losing Cell, Gene Therapy Leadership As Workforce Culling Continues

The short, strange history of gene de-extinction