Moving away from a one-size-fits-all approach, the U.S. FDA's CBER released details Jan. 11 about how it’s leveraging its growing experience with cell and gene therapies (CGTs) to exercise greater regulatory flexibility in chemical, manufacturing and control requirements for the products.
It was a battle of the companies with drugs for transthyretin-mediated amyloidosis on the first day of the J.P. Morgan 2026 Healthcare Conference with Alnylam Inc., Bridgebio Pharma Inc. and Pfizer Inc. all presenting at the annual kickoff conference. One company disclosed 2025 sales as well as 2026 revenue guidance, the second only looked back, while the third was too big to do either for a specific drug.
With rumors regarding a couple of potential mega-mergers making the rounds, the week of the annual J.P. Morgan Healthcare Conference kicked off with the official disclosure of some billion-dollar collaborations, leading with Abbvie Inc.’s exclusive licensing deal with Remegen Co. Ltd. for PD-1/VEGF-targeted bispecific antibody RC-148.
FGFR1 is a receptor tyrosine kinase that drives multiple intracellular signaling pathways involved in tumor cell proliferation, survival and progression. Because these pathways are frequently hyperactivated in colorectal cancer (CRC), FGFR1 represents a biologically relevant therapeutic target, and its inhibition has the potential to simultaneously suppress several oncogenic mechanisms.
Over the course of the year, and continuing into the latest scientific meetings, an extraordinary breadth of new antibody-drug conjugate (ADC) designs was reported, with innovations spanning targets, linkers, payloads, conjugation chemistries and overall architectures. Once defined by a simple “one target, one payload” model, the field is lately expanding into a more versatile and diverse therapeutic space.
Venetoclax has shown good results for adult acute myeloid leukemia (AML) in combination with azacitidine, but there is increasing evidence of inherent and acquired resistance. High expression of nicotinamide phosphoribosyltransferase (NAMPT) has been associated with cancer aggressiveness and poor prognosis due to increased nicotinamide metabolism.
Over the course of the year, and continuing into the latest scientific meetings, an extraordinary breadth of new antibody-drug conjugate (ADC) designs was reported, with innovations spanning targets, linkers, payloads, conjugation chemistries and overall architectures. Once defined by a simple “one target, one payload” model, the field is lately expanding into a more versatile and diverse therapeutic space.
Shenzhen Grit Biotechnology Co. Ltd. and Shanghai Vitalgen Biopharma Co. Ltd. recently presented their work to develop and evaluate a novel anti-CD19 in vivo CAR T candidate, named GT-801.
The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. Researchers from the Nara Medical University and collaborating institutions presented a potential therapeutic approach for AVWS.
Sickle cell disease (SCD) is an inherited hemoglobinopathy caused by a mutation in the gene encoding β-globin that results in hemoglobin S polymerization, red blood cell (RBC) sickling and hemolytic anemia, among others.
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