FDA Approvals - November 2025
Mitochondrial disease and mutant leukemias signal where specialty pharma is placing its biggest bets.
Affinia closes $40m Series C: CEO Rick Modi explains how low-dose gene therapy capsids could cure cardiomyopathy
“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.
Jefferies Healthcare Conference 2025 - Onyx Roundup | London Life Sciences Week 2025
Capital is available, but only for companies with clean data, clear differentiation, and a believable commercial path.
Onyx @ Jefferies: Lubor Gaal, CFO, Circio
A fully funded pharma study plus multiple R&D collaborations signals rising strategic pull.
Onyx @ Jefferies: David Chang, CEO, Allogene
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).
Onyx @ Jefferies: Jon Rees, CEO, MitoRx
The British biotech recently secured a £5.5 million pre-Series A round to advance their lead asset, Myo-004, toward clinical candidate selection.
Onyx @ Jefferies: Ali Pashazadeh, CEO, Treehill Partners
"85% of failures are us having failed the molecule, not the molecule having not delivered."
Onyx @ Jefferies: Warner Biddle, CEO, Kyverna Therapeutics
Fresh momentum from Jefferies and a new $150 million non-dilutive financing has extended its runway into 2027.
Onyx @ Jefferies: David Hung, CEO, Nuvation Bio
Nuvation is gaining real momentum after its first FDA approval, with IBTROZI now treating a growing number of patients with ROS1-positive lung cancer.
How Epicrispr are leading the first epigenetic editing trial for muscular dystrophy
“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.
Four years of progression-free survival: Nuvation Bio wins FDA approval for lung cancer
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.
FDA approvals - September 2025
September’s FDA approvals signaled shifting rules in biopharma - faster launches, smarter formulations, and bold plays from rising innovators and the familiar giants.
From stealth to spotlight: with $70m Series A, ‘total allergic control’ is Excellergy's goal
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.
New CEO of Kindbody David Stern is in the business of building families
“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.
A Legend in the making: how Ying Huang is scaling CAR-T to global markets
“After just one single infusion, about a third of patients remained in remission for five years. Some physicians even called it a functional cure,” Ying Huang underlines why CARVYKTI is reshaping expectations in oncology.