Homology Medicines Anticipating Gene Therapy Data Updates in PKU, Hunter Syndrome
Initial data from the trials of HMI-103 and HMI-203 are expected in 2023.
Cilta-cel Garners More Positive Data in Early and Late Relapsed Multiple Myeloma Treatment
Ying Huang, chief executive officer, Legend Biotech, discussed new data from the CARTIFAN-1 and CARTITUDE-2 studies.
Phase 2 Sepsis Cell Therapy Trial Cleared to Expand into France and Belgium
Allocetra previously demonstrated efficacy in a phase 1b trial for patients with sepsis.
Jakub Svoboda, MD, on Promising Efficacy With huCART19-IL18 in NHL, CHL
The associate professor of medicine from University of Pennsylvania discussed updated data on huCART19-IL18 presented at the ASH 2022 meeting.
Around the Helix: Cell and Gene Therapy Company Updates – January 4, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
BCMA-directed CAR-T Gets IND Clearance for R/R Multiple Myeloma
CT103A is currently being evaluated in multiple clinical trials in China.
Swati Naik, MBBS, on Investigating CD123-CAR T Therapy for Relapsed AML
The assistant member at St. Jude Children’s Research Hospital discussed updated data from a phase 1 study being conducted at the hospital.
Cellectis Shifts to In-House Manufacturing With UCART22
The BALLI-01 study, initiated in 2019, has now dosed its first patient with an in-house manufactured CAR T-cell therapy candidate.
Tessa Therapeutics Shifts Focus to Allogeneic Therapies
The company is seeking out other companies to continue development of its autologous CAR-T platform.
BCMA/CD19 CAR T Shows Efficacy in Newly Diagnosed Multiple Myeloma
Updated data were presented at the ASH 2022 annual meeting.
Real-World Axi-Cel Data Show QOL Improvements in LBCL
The first year after treatment yielded statistically significant improvements in overall QOL and symptoms within the first year of treatment for relapsed/refractory large B-cell lymphoma
Liso-Cel Confirms Superiority Over SOC in Second-Line LBCL
Second-line lisocabtagene maraleucel reduced the risk of an event occurring by 64.4% compared with standard-of-care chemoimmunotherapy induction and autologous stem cell transplantation.
2022 Year in Review: Top News and Insights in Cell Therapy
Review some of our most-viewed coverage of advancements in cell therapies, including study data and clinical trial updates.
Fidanacogene Elaparvovec Bests Standard of Care in Phase 3 Hemophilia B Study
Annualized bleeding rates were superior with the gene therapy fidanacogene elaparvovec compared with factor IX treatment for patients with moderately severe to severe hemophilia B.
BAFFR-targeted CAR-T Demonstrates Complete Response in First Patient With B-NHL in Phase 1 Trial
The CR was achieved at 1-month post-treatment.
2022 Year in Review: Top News and Insights in Gene Therapy
Review some of our most-viewed coverage of advancements in gene therapies, including study data and clinical trial updates.
"Remote-Controlled" CAR-T Technology Can Help Personalize Cell Therapy for Cancer
Crystal Mackall, MD, an Alliance for Cancer Gene Therapy scientist, is helping create a remote-control technology to personalize CAR T-cell therapy for each cancer patient.
Armin Ghobadi, MD, on Helping Expand CAR T Cells With NT-I7
The associate professor of medicine at Siteman Cancer Center and WUSTL discussed updated data from a study of NT-I7 and tisa-cel presented at ASH 2022.
CGTLive’s Weekly Rewind – December 23, 2022
Review top news and interview highlights from the week ending December 23, 2022.
Gene Therapies Advance in Danon Disease, CLN2 Batten Disease
Both REGENXBIO and Rocket Pharmaceuticals have announced trial updates in their gene therapy programs for rare diseases.
Around the Helix: Cell and Gene Therapy Company Updates – December 21, 2022
Solid Tumor CAR T Collaboration Cleared for Trials
Invectys and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience, announced the therapy and collaboration in June 2022.
Ide-cel Shows Efficacy in Early Relapsed Multiple Myeloma
Patients had an ORR of 83.8% with a median DOR of 15.7 months in data presented at the ASH 2022 meeting.
Radiation-Induced Xerostomia Gene Therapy Shows Promise in Phase 1 Trial
No dose-limiting toxicities or treatment-related serious adverse events were reported among 24 treated patients.
Running With Scissors: The FDA Moves to Tame Gene Therapy Drug Development
With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.
Steven Pipe, MD, on Continuing Durability of Etranacogene Dezaparvovec in Hemophilia B
The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed new follow-up data from the HOPE-B study presented at ASH 2022.
FDA Approves Ferring's Gene Therapy for Bladder Cancer
The approval follows a CRL that was issued in 2021 due to problems with chemistry, manufacturing, and controls.
CGTLive’s Weekly Rewind – December 16, 2022
Review top news and interview highlights from the week ending December 16, 2022.
B-VEC Continues to Show Durable Wound Healing in Dystrophic Epidermolysis Bullosa
Half of wounds treated with the gene therapy had complete healing at both months 3 and 6 compared to 7% of placebo-treated wounds.
Sham Mailankody, MBBS, on Updates on ALLO-715 CAR T in R/R Multiple Myeloma
The associate attending physician at Memorial Sloan Kettering Cancer Center discussed updated data presented at the ASH 2022 meeting.
