Gene Therapies

With its lead gene therapy nearing pivotal data, enGene installs a new chairman with a track record of commercial success, signaling a critical shift from R&D to market readiness.

As Corrixr's novel CRISPR therapy nears human trials, the appointment of Seagen veteran Nancy Whiting signals a strategic shift from lab to market.

RNAi pioneer Alnylam unveils promising data for its gene-silencing drugs, targeting Alzheimer's and CAA at their genetic source and offering new hope.

Voyager Therapeutics' new gene therapy just cleared a key FDA hurdle. With promising preclinical data, it's a major momentum signal in the fight against Alzheimer's.

Ocugen inks a potential $255M+ deal to bring its OCU400 gene therapy for Retinitis Pigmentosa to the MENA region, a strategic move into a high-need market.

A mining firm just secured a key gene therapy patent for ALS. Timothy Bell dissects the audacious strategy of funding cutting-edge biotech with Greenland's mineral wealth.

For children with the rare metabolic disorder MMA, a new gene therapy offers hope. Preclinical data reveals a potential one-time fix with lifelong effects.

With €33M in fresh capital, Cyllene Therapeutics is advancing a novel gene therapy for bladder disease. We analyze the tech, the investors, and the plan.

Nanoscope's revolutionary MCO-010 gene therapy offers hope to millions with retinal disease. A new partnership with Catalent is the critical final step.

With €33M in new funding, Cyllene Therapeutics is advancing a novel gene therapy using HSV-1 vectors. Could this be the one-shot cure for millions?

4D Molecular Therapeutics is set to reveal 2-year data on a potential one-time gene therapy for wet AMD, a move that could end the relentless cycle of eye injections.

With a new CFO experienced in biotech financing, Tenaya is shoring up its leadership to guide its cardiovascular gene therapies toward pivotal trials.

4DMT secures a complex $200M credit facility, choosing strategic debt over equity. We dissect the deal and what it signals for late-stage biotech financing.

uniQure's upsized $259M offering signals massive investor confidence, fueling its Huntington's therapy, AMT-130, and reshaping the gene therapy landscape.

A new wave of disease-modifying therapies is poised to ignite the Huntington's disease market, projecting 14% annual growth and turning a field of despair into a high-stakes biotech race.

An independent safety board just gave Medera's novel gene therapy for a stubborn form of heart failure a major endorsement, de-risking its path to the FDA.

Hatch BioFund's new investment in Princeton spinout Optimeos funds a novel nanoparticle platform that could solve one of medicine's biggest challenges.

After game-changing FDA news for its Huntington's therapy, uniQure's upsized $225M offering signals massive investor confidence and accelerates its mission.

Decades-old treatments for bladder cancer are failing. Now, a wave of gene and viral therapies is sparking a market revolution and offering new hope.