Latest

SynaptixBio says rare disease biotech investment is increasing in the US following renewal of the FDA's Priority Review Voucher program, while UK-based rare disease companies continue to lack an equivalent incentive and remain dependent on public markets, private investment, or acquisition to secure capital.

Today’s BioPharm Brief covers rare disease investment, an FDA fast track designation for a Parkinson disease therapy, and phase 2 results for an siRNA treatment in polycythemia vera.

Dr Steve Harr, Sana Biotechnology's CEO, identifies scalability, commercialization, and reimbursement as the defining hurdles for curative islet cell therapy in type 1 diabetes.

Silence's siRNA drug freed 88% of trial patients from ongoing phlebotomies, a chronic burden for people with polycythemia vera.

Stand Up To Cancer has awarded its Nina Nicolai Pancreatic Cancer Innovation in Collaboration Award to a research team developing a combination approach that pairs an engineered mRNA vaccine with KRAS G12D-directed TCR-T cell therapy, aiming to sustain antigen production and improve T-cell responses against pancreatic cancer.

Early Parkinson patients could gain a first disease-modifying option as ACI-7104 clears a major FDA hurdle after strong interim results.

Today’s BioPharm Brief looks at Akeso’s bispecific ADC and ivonescimab combination in breast cancer, Novo Nordisk’s AI partnership with AWS, and Ascletis’ once-monthly obesity candidates.

Ascletis has initiated two Phase I studies for its obesity pipeline: ASC36, a once-monthly amylin receptor peptide agonist, and ASC36_35FDC, a once-monthly fixed-dose co-formulation of ASC36 with its GLP-1R/GIPR dual agonist ASC35, following recent FDA IND clearances for both candidates.

Bristol Myers Squibb has selected Houston's Generation Park for a new $2.3 billion multi-modal manufacturing campus capable of producing small molecules, biologics, and antibody-drug conjugates, adding to a growing wave of large-scale US pharmaceutical manufacturing investment.

AWS's AI and cloud tools aim to compress Novo Nordisk's path from drug target to first human dose for chronic disease patients.

This week’s BioPharm Brief looks at FDA Fast Track designation for BioInvent’s BI-1808, Neurocrine’s Phase 1 obesity study of a GLP-1/GIP/glucagon triple agonist, and Replimune’s accelerated approval for Tudriqev plus nivolumab in advanced melanoma.

Moderna's mFLUSIVA approval offers a regulatory case study for mRNA vaccine sponsors, covering comparator selection lessons from the FDA's reversed refusal-to-file, competitive implications for Pfizer and Sanofi's mRNA flu programs, and what the accelerated approval pathway means for long-term regulatory risk.

BI-1808 plus pembrolizumab posted a 24% response rate in platinum-resistant ovarian cancer, tripling pembrolizumab's historical rate alone.