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.
|Articles|December 26, 2016
Year in Review: Patients Recount Their Personal Battles with Specialty Diseases
Author(s)Lauren Santye, Assistant Editor
Patients share their personal stories with Specialty Pharmacy Times.
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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.
October’s hematology coverage highlighted first-line pirtobrutinib, questions about myeloma treatment duration and early intervention, and pharmacists’ roles in medication safety and access.
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