Gene Therapies

With its lead drug for a rare blood cancer nearing European submission, Cellectar Biosciences advances its targeted radiotherapy platform on two fronts.

Immunome announces stellar Phase 3 data for its desmoid tumor drug, varegacestat, and details a robust, well-funded ADC pipeline aimed at solid tumors.

With promising clinical data for its heart disease treatments, Lexeo Therapeutics aims to capture investor attention at the upcoming Leerink conference.

A new trial for a cell therapy targeting a rare eye disease offers hope, driven by a patient-advocate's journey and with potential to treat common AMD.

Peli BioThermal and Polar Group partner to upgrade Brazil's pharma logistics, tackling a booming market and ensuring safer delivery of advanced medicines.

As SAB Biotherapeutics prepares for key investor talks, a closer look reveals its novel T1D therapy and the unique bovine tech aiming to change treatment.

After a major turnaround, Genetix Biotherapeutics announces its first profitable quarter and plans to double production of its high-stakes gene therapies.

Fresh off a major clinical trial victory, Alumis Inc. heads to a key investor conference. What's next for its promising pipeline and soaring stock?

For millions suffering from relentless chronic cough with no approved treatments, Trevi Therapeutics' Haduvio offers a new possibility backed by strong clinical data.

Lynk Pharmaceuticals reports its oral drug zemprocitinib met all goals in a late-stage study for atopic dermatitis, offering rapid itch relief and a strong safety profile.

As MSA Awareness Month begins, a novel drug from Alterity Therapeutics shows significant promise in slowing the devastating and untreatable neurodegenerative disease.

With a strong cash runway and pivotal trials underway, Sionna Therapeutics aims to revolutionize CF treatment with a novel class of NBD1 stabilizer drugs.

With positive Phase 3 data for its first-in-class Stargardt disease drug and a massive new financing round, Belite Bio pivots toward commercialization.

A new partnership is building an ultra-low temp supply chain, paving the way for the first off-the-shelf stem cell therapies for pets with chronic disease.

Born from a Novartis mega-deal, Atrium Therapeutics aims its novel RNA platform at rare, untreatable genetic heart diseases. Will its strategy succeed?

A new oral therapy, mavorixafor, receives a key recommendation in Europe, offering a beacon of hope for patients with a debilitating ultra-rare immune disorder.

After a strategic pivot to focus purely on cell and gene therapy, BioLife reports strong revenue growth and a profitable quarter, eyeing further gains in 2026.

GEMMABio has dosed the first infant in a trial for its next-gen SMA1 gene therapy, a new approach designed to improve safety and global access.

Sarepta will unveil crucial long-term data for its Duchenne gene therapy and exon-skipping drugs at MDA 2026, a pivotal moment for patients and the company.

With the hire of Greg Crescenzi, the famed academic repository signals a major strategic expansion to bridge the gap between scientific sharing and drug development.