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.
Vutrisiran Approved for hATTR Amyloidosis
The approval came earlier than the recently extended PDUFA date of July 24, 2022.
CAR T-Cell Therapy Yields High Response Rates in Follicular Lymphoma
Mustang Bio presented data on MB-106 at the 2022 EHA Congress.
Tisa-Cel Shows Durable Efficacy in Pediatric and Young Adult Patients With R/R B-Cell Acute Lymphoblastic Leukemia
Among treated patients, 42% achieved 5-year event free survival.
Around the Helix: Cell and Gene Therapy Company Updates – June 15, 2022
Re-Dosing With Gene Therapies in Rare Diseases
Takashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.
Dosing Continues in Hemophilia B Gene Therapy Trial
Updated date from the B-LIEVE study is expected in the second half of 2022 and first half of 2023.
Clinical Hold Lifted on Homology’s pheNIX Trial for Phenylketonuria
The trial was placed on hold after elevated liver function test scores.
Massimo Trucco, MD, on Improving Access to Islet Cell Transplant
The director of the Alleghany Health Network Institute of Cellular Therapeutics discussed islet cell transplant for the treatment of chronic pancreatitis.
Dual-Targeted CAR T-Cell Therapy Continues to Show Efficacy in R/R Multiple Myeloma
GC012F also showed efficacy in B-cell non-Hodgkin lymphoma.
Anbal-Cel Shows Promising Responses in Large B-Cell Lymphoma
The CD-19 CAR T-cell therapy is developed using Curocell’s OVISTM platform.
Exa-Cel Eliminates Transfusions in TD Thalassemia and VOCs in Sickle Cell Disease
Results from the CLIMB THAL-111 and CLIMB SCD-121 trials were presented at the EHA 2022 Congress.
Axi-Cel More Efficacious With Higher Toxicity Than Tisa-Cel in Diffuse Large B-Cell Lymphoma
Response rates were significantly higher but patients had more cases of ICANS and CRS with axi-cel treatment compared to tisa-cel treatment.
Lentiviral CAR T Therapy Delivers Good Efficacy, Safety in R/R Multiple Myeloma
Participants in the trial achieved a sustained response and experienced low-grade toxicity.
CD30-Targeted CAR T Cell Therapy Promising in Heavily Pretreated Hodgkin Lymphoma
A phase 2 clinical trial evaluating a T-cell memory enriched anti-CD30 CAR-T is now underway.
