Onyx @ Jefferies: Ali Pashazadeh, CEO, Treehill Partners
"85% of failures are us having failed the molecule, not the molecule having not delivered."
"85% of failures are us having failed the molecule, not the molecule having not delivered."
Capital is available, but only for companies with clean data, clear differentiation, and a believable commercial path.
A fully funded pharma study plus multiple R&D collaborations signals rising strategic pull.
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).
“We’re really on the precipice of moving the company into that next stage of maturity,” Dr. Mike Exton
Nuvation is gaining real momentum after its first FDA approval, with IBTROZI now treating a growing number of patients with ROS1-positive lung cancer.
The British biotech recently secured a £5.5 million pre-Series A round to advance their lead asset, Myo-004, toward clinical candidate selection.
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.
Fresh momentum from Jefferies and a new $150 million non-dilutive financing has extended its runway into 2027.
Mitochondrial disease and mutant leukemias signal where specialty pharma is placing its biggest bets.
“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.
“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.
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.
September’s FDA approvals signaled shifting rules in biopharma - faster launches, smarter formulations, and bold plays from rising innovators and the familiar giants.
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.