Paulina Velasquez, MD, on Finding New Targets for Cell Therapy
The assistant member at St. Jude Children’s Research Hospital discussed the ASH 2022 basic and translational science session she moderated.
Crigler-Najjar Syndrome Gene Therapy Trial Enters Pivotal Portion
Some patients previously treated with GNT-003 were able to maintain the stoppage of phototherapy for at least 1 year.
Second Generation MUC1-CAR T Therapy Cleared for Solid Tumor Trial
huMNC2-CAR22 is designed to avoid off-tissue expression and reduce T-cell exhaustion.
Val-rox Meets End Points in Hemophilia A at 3 Years
The new analysis data was requested by the FDA ahead of the therapy’s March 31, 2023 PDUFA date.
Canavan Disease Gene Therapy Continues to Show Promise
No serious adverse events related to the gene therapy have been reported.
Celularity’s MSC Platform Shows Clinical Benefit for Crohn Disease in Legacy Analyses
Phase 1/2 studies evaluated PDA001 in 50 participants with CD.
Leber Congenital Amaurosis-2 Gene Therapy Trial Doses First Patient
The company expects to provide initial data from the trial in the second half of this year.
Developing Nonviral Gene Therapy for Hemophilia A
Brent Warner, president, gene therapy, Poseida Therapeutics, discussed data on the preclinical P-FVIII-101 presented at ASH 2022.
First Patient Dosed in Treg Cell Therapy Trial for Myelofibrosis
CKO804 is being evaluated as an add-on therapy to ruxolitinib in patients whose disease had a suboptimal response to ruxolitinib alone.
MPS-IH HSC Gene Therapy Gets IND Clearance
Promising results were previously reported from a proof-of-concept study involving 8 patients.
Sickle Cell Disease Gene-Editing Therapy Trial Voluntarily Paused Following Serious Adverse Event
The first patient dosed in the study experienced a case of prolonged pancytopenia that has required ongoing transfusion and growth factor support.
CGTLive’s Weekly Rewind – January 6, 2023
Review top news and interview highlights from the week ending January 6, 2023.
Intellia to Advance Development of ATTR Amyloidosis, Angioedema, AATD Gene Therapies
The company is kicking off 2023 by announcing planned advancements for several gene therapies in its pipeline.
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.
