Gene Therapies

The specialty infusion provider is the first to earn a rigorous new distinction, signaling a major leap forward in patient safety and quality of care.

Ratio Therapeutics appoints Dr. Colin Hayward as CMO, a strategic move to advance its sarcoma trial and challenge leaders in the booming radiopharmaceutical market.

Positive Phase 3 results for BBP-418 offer a potential first-ever treatment for LGMD2I/R9, a progressive muscle disease with no current therapies.

A novel compound, IRX4204, shows promise in preventing triple-negative breast cancer in lab studies, offering new hope for a safe, effective preventative.

With a strong cash position and new leadership, Artiva pivots its AlloNK® cell therapy to autoimmune disease, targeting a pivotal trial for RA in 2026.

With its CAR-T therapy achieving franchise profitability and treating 10,000 patients, Legend Biotech eyes overall profit and next-gen cancer treatments.

A global consortium pioneers new drugs designed specifically for children, moving away from harsh adult treatments for aggressive pediatric cancers.

A new line of ultra-pure proteins with endotoxin levels 10x below the standard could revolutionize advanced therapies and fix a key source of error in science.

After 15 years without new therapies, Tonix Pharma's TONMYA enters the fibromyalgia market with a unique sublingual drug and an ambitious commercial plan.

A new, highly selective drug for Duchenne muscular dystrophy shows potential to be safer and more effective, targeting both muscle and heart decline.

IDEAYA Biosciences begins a key trial for its targeted therapy IDE892, aiming to conquer hard-to-treat tumors and pivoting to focus on its proprietary assets.

Early access programs for a rare blindness therapy in France, Israel, and the US offer hope to patients and provide a crucial financial lifeline for the company.

A new trial in China tests a dual-action antibody for esophageal cancer, a top killer. The therapy aims to unleash a two-pronged immune attack on tumors.

After its public debut, BBOT unveils exciting early data from three unique cancer drugs, including a first in pancreatic cancer, backed by a strong cash runway.

With a strong cash position, ArriVent pushes its lead lung cancer drug toward a mid-2026 data readout while building a new pipeline in ADCs.

New preclinical data shows Quanta Therapeutics' inhibitors remain potent against cancers that have developed resistance to advanced pan-RAS treatments.

With a melanoma drug on track for 2027 and a deep pipeline, Immatics' 2025 net loss is part of a larger strategy to dominate PRAME cancer therapy.

Biotech firm Immunis, Inc. secures a promising drug candidate to treat a debilitating side effect of Parkinson's therapy, offering hope to millions.

With strong revenue growth and critical FDA decisions on the horizon, MannKind's leadership prepares to outline its vision for investors in Miami.

After solving a key manufacturing flaw, CervoMed advances its promising dementia drug, neflamapimod, to a Phase 3 trial with an optimized dose.