Gene Therapies

Drs. Zoghbi and Bird win the prestigious Horwitz Prize for turning Rett syndrome from an untreatable mystery into a reversible condition, offering new hope.

ElevateBio's new COO, Steven Falcone, is a strategic weapon against the crushing cost of advanced therapy manufacturing. This isn't just a hire; it's a war on the price of miracles.

For families facing the grim prognosis of GM2 gangliosidoses, a new alliance offers a glimmer of hope, revealing the strategic interplay of academia and industry.

Abeona Therapeutics celebrates rising sales for its life-changing RDEB therapy, ZEVASKYN. But behind the numbers lies a story of manufacturing hurdles and mounting debt.

Genomic pioneer Sangamo sells key assets to PTC and Lilly in a $163M bankruptcy deal, reshaping the future of gene therapy and rare disease treatment.

Metagenomi Therapeutics is on track for a pivotal 2027 clinical trial, but its advanced gene editing tech faces a market wary of high-cost cures.

With its Duchenne therapy nearing a pivotal FDA meeting, Solid Biosciences isn't just developing a drug; it's building a network for genetic repair.

Cryoport hits a key profitability milestone, but the real story is its tightening grip on the booming gene therapy market. Is this a true turning point?

Phoenix Nest secures a pivotal $3M NIH grant to manufacture a potential cure for Sanfilippo syndrome, a fatal childhood disease with no current treatment.

Soprano Eleonora Buratto lends her voice to fight 'butterfly disease,' highlighting an innovative venture philanthropy model rewriting the playbook for rare illnesses.

Editas Medicine’s new board member signals a sharp focus on cardiovascular disease, a strategic pivot that could reshape the competitive gene editing race.

Immix Biopharma heads to Morgan Stanley's key conference. Can its promising CAR-T therapy for AL Amyloidosis convince investors to fund the final mile?

The first Animal Longevity Summit marks a pivotal moment, as research into pet aging and biotech investment converge to create a new frontier in healthspan.

A single IV shot to halt Parkinson's? Shape Therapeutics' radical RNA-editing therapy, now backed by The Michael J. Fox Foundation, aims to do just that.

Oak Hill Bio is betting on a therapy shelved by a pharma giant. Its new trial for Angelman syndrome tests a disruptive model for rare disease innovation.

An upcoming summit for LGMD isn't just another conference. It's a key signal that patient advocacy, biotech innovation, and regulatory shifts are converging.

Pacira BioSciences de-risks its promising knee osteoarthritis gene therapy by solving the manufacturing puzzle, a key step from lab to market for millions.

Beam Therapeutics is set to unveil pivotal data for its AATD gene editor. Success could transform a disease and validate a multi-billion-dollar platform.

With key hires in finance, regulatory, and manufacturing, Cyllene Therapeutics signals it's preparing for the big leagues. But can it deliver?

A new report reveals a seismic shift in drug manufacturing. Forget massive factories; the real power now lies with a handful of specialists building our medical future.