Clinical results reveal new opportunities and setbacks in rare disease and cancer care, shaping treatment strategies and future regulatory decisions.
News|Articles|October 9, 2025
Streamline AAV-based Gene Therapies with High-Performing Off-the-Shelf Plasmids
Author(s)Catalent
Optimized plasmids are vital for viral vector production. Catalent’s off-the-shelf plasmids help AAV gene therapy developers enhance efficiency, reduce costs, and improve consistency across development and manufacturing.
Related to this article
Phase 3 interim data for RYZ101, the lead asset from Bristol Myers Squibb’s $4.1 billion RayzeBio deal, meet the primary endpoint.
Efzimfotase alfa missed significance on the six-minute walk test in adolescents and adults, while the FDA review of the therapy proceeds toward 2027.
Odyssey's 146-patient phase 2a trial will pair OD-001 with vedolizumab, targeting both innate and adaptive immune drivers of ulcerative colitis.
MT027, T-MAXIMUM's B7-H3 CAR-T therapy, earns a second FDA-cleared phase 2 trial, now testing lung and breast cancer brain metastases.
New clinical data highlight improved disease control, reduced eye bulging, and weight-loss benefits, potentially broadening options for patients with unmet needs.
Antisense oligonucleotides are reshaping drug design, from RNase H gapmers to inhaled delivery; this FAQ explains how they work and why trials still fail.
Bristol Myers Squibb's Nathan Pennell explains how the MRD endpoint supports accelerated approval in multiple myeloma without changing how clinicians monitor patients.