Driven by the precision of viral biology, 4D Molecular Therapeutics is redefining gene therapy’s reach - scaling a once ultra-rare field into broad, sustainable markets.
Published 3 Dec 2025
Mitochondrial disease and mutant leukemias signal where specialty pharma is placing its biggest bets.
Published 2 Dec 2025
“Over the past year, we have made tremendous progress…” Rick Modi says, as Affinia Therapeutics pushes its BAG3-DCM program toward first-in-human testing, aiming to prove that next-generation capsids can restore heart function at dramatically lower doses.
Published 26 Nov 2025
Capital is available, but only for companies with clean data, clear differentiation, and a believable commercial path.
Published 19 Nov 2025
A fully funded pharma study plus multiple R&D collaborations signals rising strategic pull.
Published 18 Nov 2025
Cema-cel is already in the pivotal Phase 2 ALPHA3 frontline lymphoma trial, with manufacturing capacity sized for tens of thousands of annual doses (up to ~60,000 across products).
Published 18 Nov 2025
The British biotech recently secured a £5.5 million pre-Series A round to advance their lead asset, Myo-004, toward clinical candidate selection.
Published 18 Nov 2025
"85% of failures are us having failed the molecule, not the molecule having not delivered."
Published 17 Nov 2025
Fresh momentum from Jefferies and a new $150 million non-dilutive financing has extended its runway into 2027.
Published 17 Nov 2025
Nuvation is gaining real momentum after its first FDA approval, with IBTROZI now treating a growing number of patients with ROS1-positive lung cancer.
Published 17 Nov 2025
Scarred lungs and hot flashes are two FDA wins for specialty pharma.
Published 5 Nov 2025
“We can regulate any gene, anywhere,” says CEO Dr. Amber Salzman, whose Epicrispr team is pioneering epigenetic editing - a precise, non-cutting approach - to transform neuromuscular disease treatment through durable, targeted control of gene activity.
Published 4 Nov 2025
With a median progression-free survival nearing four years in ROS1 lung cancer, Nuvation Bio’s breakthrough therapy signals a turning point for precision oncology - validating Dr. David Hung’s patient-first vision.
Published 21 Oct 2025
September’s FDA approvals signaled shifting rules in biopharma - faster launches, smarter formulations, and bold plays from rising innovators and the familiar giants.
Published 15 Oct 2025
Backed by $70 million in Series A funding, Excellergy emerges from stealth with a mission to lead a new era in immunology - targeting the full IgE pathway to redefine how allergic diseases are treated.
Published 13 Oct 2025
“Fifteen to twenty years ago, a woman under 35 had a 20% chance of live birth per IVF cycle. At Kindbody, today, it’s greater than 50%,” David Stern credits lab innovation and AI for rewriting fertility’s odds.
Published 9 Oct 2025
London · 15–19 November 2026
Explore Onyx’s independent guide to the events and make the most of your week in London.
