Connecting Patient Advocacy and Industry for Gene Therapy Development
Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the panel discussion she participated in at BIO 2022.
Pursuing Slow but Steady Gene Therapy Development
Michael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.
Sarepta’s Duchenne Muscular Dystrophy Gene Therapy Placed on Clinical Hold
The hold was related to a serious adverse event of hypomagnesemia in an ongoing phase 2 clinical trial.
Late-Onset Pompe Disease Gene Therapy Trial Placed on Clinical Hold
The hold comes after a serious AE of peripheral sensory neuropathy.
David Weinstein, MD, on Delivering Gene Therapy Directly to the Brain
The senior vice president of clinical development at Passage Bio discussed intracisternal magna administration of PBGM01.
Nilanjan Ghosh, MD, PhD, on Real-World Efficacy of CAR T-Cell Therapies
The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed data from real-world experience studies.
Liso-Cel Approved for Second-Line Large B-Cell Lymphoma
The approval builds off of liso-cel's (Breyanzi) previous indication for third-line or later treatment in LBCL.
CGTLive’s Weekly Rewind – June 24, 2022
Review top news and interview highlights from the week ending June 24, 2022.
Val-Rox Approval Favored in Europe, Stalled in US
BioMarin received a positive CHMP opinion after the FDA delayed val-rox's BLA filing for the second time.
Krystal Seeks Approval for Dystrophic Epidermolysis Bullosa Gene Therapy
B-VEC recently showed efficacy in the phase 3 GEM-3 trial.
Steven W. Pipe, MD, on Challenges in Gene Therapy Trials for Hemophilia
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed common challenges with gene therapy clinical trials.
Huntington Disease Gene Therapy Promotes Reduction in Mutant Huntington Protein, Neurofilament Light
NfL peaked 1-month post-treatment as expected and returned to near baseline at 12 months.
Gene-Mediated Immunotherapy for Ovarian Cancer Clears Safety Hurdle, Will Continue Dosing
The phase 1/2 OVATION 2 study is expected to complete enrollment by the third quarter of this year.
Thomas Martin, MD, on Exciting Cell Therapy Options in Multiple Myeloma
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed cilta-cel and upcoming cell therapies.
Around the Helix: Cell and Gene Therapy Company Updates – June 22, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Reinfusion of CAR T Therapy Shows Promise in B-ALL
Long term survival was limited and all patients with B-cell acute lymphoblastic leukemia eventually relapsed.
Hemophilia B Gene Therapy Well-Tolerated, Reduced Need for FIX Replacement Therapy
Annualized bleeding rate fell to 0 at a median of 58 weeks after infusion.
Massimo Trucco, MD, on the Potential of Islet Cell Transplant
The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed possible applications of the procedure.
Children Treated With Zolgensma for Presymptomatic SMA Achieve Age-Appropriate Motor Milestones
All children in the phase 3 SPR1NT study achieved the primary end point of independent sitting.
Developing Invariant Natural Killer T Cells for Multiple Myeloma, GVHD
Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed different indications for the iNKT therapy AgenT-797.
Keith Fargo, PhD, on Potential Treatment Avenues for Charcot-Marie-Tooth
The chief scientific officer of the CMT Research Foundation discussed investigative cell and gene therapy approaches for treating CMT.
IND Cleared for γδ T-Cell Therapy in Non-Hodgkin Lymphoma
ACE1831 will soon be evaluated in a phase 1, first-in-human, clinical trial.
Adriana Rossi, MD and Luciano J. Costa, MD, PhD on Earlier CAR T in Multiple Myeloma
Oncologist discuss the possibility of earlier-line CAR T-cell therapies in MM.
Noopur Raje, MD, on Current CAR T Options in Multiple Myeloma
The director of the Center for Multiple Myeloma at Mass Gen discussed current CAR T-cell therapies in multiple myeloma.
NT-I7 Boosts CAR T-Cell Expansion, Persistence, and Anti-Tumor Activity in Preclinical Study
A multisite phase 1 clinical trial for NT-I7 is underway in patients with relapsed/refractory large B-cell lymphoma.
CGTLive’s Weekly Rewind – June 17, 2022
Review top news and interview highlights from the week ending June 17, 2022.
Developing Controllable Tumor Infiltrating Lymphocyte Therapies
Jan ter Meulen, MD, PhD, chief scientific officer, Obsidian Therapeutics, discussed the potential of the cytoDRIVE platform.
T-Charge CAR T-Cell Therapy Shows Efficacy in Multiple Myeloma
The therapy is now being evaluated in a phase 2 study.
CTX130 Shows Acceptable Safety Profile, Clinically Meaningful Responses in R/R T-Cell Lymphoma
CTX130 is also being investigated for the treatment of r/r renal cell carcinoma in a phase 1 clinical trial called COBALT-RCC (NCT04438083).
Frederick Locke, MD, on Further CAR T Research
The vice-chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center discussed more work to be done with CAR T-cell therapies.
