5-Year Follow-Up Data For CPX-351 in AML

If gene therapies are expected to deliver decades of benefit, long-term evidence collection must be coordinated, useful to patients, and financially sustainable.

Matthew Porteus, MD, PhD, highlighted preclinical advances in automated stem cell gene editing that could improve manufacturing scalability while supporting the development of next-generation genetic medicines.

If approved, the combination would be the first chemotherapy-free, Bruton tyrosine kinase inhibitor–based regimen for adults with newly diagnosed mantle cell lymphoma (MCL).

Manufacturing delays, financial barriers, and complex care coordination can limit access to gene therapy, emphasizing the need for patient-centered programs and long-term follow-up.

Experts highlighted how Diamond-Blackfan anemia illustrates the scientific, financial, and manufacturing challenges that can prevent promising gene therapies for ultra-rare diseases from reaching patients.

Experts highlighted recent gene therapy approvals for inherited immune disorders while addressing ongoing challenges in manufacturing, long-term safety, and patient access.

At the 2026 ASTCT Gene Therapy Summit, experts highlighted how chronic pain, psychological challenges, and neurologic complications may persist despite successful treatment.

Pharmacists navigate BCMA therapy sequencing in relapsed/refractory multiple myeloma, weighing linvoseltamab logistics, community readiness, safety monitoring, and smoother care transitions.