Gene Therapies

Quoin Pharma's new data for a rare skin disease shows life-changing results in children, offering hope for families. What do the numbers really mean?

BioAge Labs starts a Phase 2 trial for BGE-102, an oral drug targeting inflammation to reduce heart risk, a strategy that could redefine cardiovascular care.

The 8th annual 'Shine the Light' campaign unites 47 groups to fight Sickle Cell Disease, but behind the red-lit landmarks lies a deeper crisis of access to new, multi-million dollar cures.

A new partnership between Matica Bio and Cirsium Biosciences aims to shatter the high-cost, closed-off world of gene therapy manufacturing. Is this the key?

Verge Labs' new AI model promises to predict brain activity from a blood draw, potentially upending the costly and failure-prone world of neurology R&D.

Bruce Eaton's appointment as Chairman at Broken String isn't just a new hire; it's a strategic move to make its INDUCE-seq technology the industry's gold standard.

New clinical trial results for XWRAP® show a significant leap in healing diabetic ulcers, positioning Applied Biologics as a formidable player in a challenging field.

Lexeo Therapeutics finalizes a pivotal trial for its Friedreich ataxia gene therapy, leveraging a novel trial design and accelerated FDA pathway.

Aussie biotech ENA Respiratory secures crucial US patents for its novel immune modulator, signaling a major strategic move in the multi-billion dollar fight against COPD.

A breakthrough publication identifies a novel protein, LanCL1, as a key target for neuropathic pain, validating a new class of drugs for millions of sufferers.

Sanofi's latest FDA nod for Tzield isn't just a new drug approval; it's a strategic shift from symptom management to disease modification in Type 1 Diabetes.

MBX Biosciences' new one-year data for canvuparatide isn't just a clinical win; it's a calculated move to dominate a billion-dollar market left vacant.

With a new CMO, Get A Drip is capitalizing on the longevity trend. But as it expands, it faces questions on scientific rigor and regulatory oversight.

A new FDA-designated device aims to filter a deadly protein from the blood of sepsis patients. Can this novel approach finally conquer a global health crisis?

A $4M grant for a Parkinson's drug isn't just charity; it's a strategic bet on a new era of medicine, where philanthropy acts as the catalyst for the next big cure.

New data on an all-oral combo therapy for AML shows remarkable efficacy, offering new hope for patients and cementing Syndax's competitive advantage.

A Swedish firm's novel cell therapy is the first of its kind in Europe, challenging the grim prognosis of pancreatic cancer and testing a new model for biotech.

A market set to triple, driven by gene-level therapies, promises a revolution for children with rare epilepsies. But can our systems deliver this hope?

At EHA 2026, Parexel isn't just presenting data; it's unveiling a bold new strategy to fix a broken system and accelerate life-saving blood cancer drugs.

One in six people globally faces infertility, yet a pervasive stigma silences their struggle. A worldwide movement is now uniting millions to demand change.