Ornithine Transcarbamylase Gene Editing Program Nabs Another FDA Designation
iECURE is planning to submit an IND for GTP-506 in mid-2023.
Astrocyte Cell Therapy Well-Tolerated in Amyotrophic Lateral Sclerosis
Postmortem analyses revealed cell survival and GDNF production in transplanted areas.
Sequential T-Cell Redirection Therapies Yield Deep, Durable Responses in R/R Multiple Myeloma
Patients receiving CAR T-cell therapy or bispecific antibody therapies as first salvage therapy had an ORR of 84%.
Around the Helix: Cell and Gene Therapy Company Updates – September 7, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
IND Halted for T-Cell Malignancy Multiplex-Edited Cell Therapy
The FDA has requested additional preclinical data from Beam Therapeutics on BEAM-201.
Tecartus Approved in Europe for R/R Acute Lymphoblastic Leukemia
Of 55 evaluable patients in ZUMA-3, 71% achieved complete remission (CR) or CR with incomplete hematological recovery (CRi).
Adam D. Cohen, MD, on Cilta-Cel for Lenalidomide-Refractory Multiple Myeloma
The director of myeloma immunotherapy at University of Pennsylvania discussed cilta-cel's efficacy in lenalidomide-refractory multiple myeloma.
Applying a Novel Gene Editing Approach to Chronic Hepatitis B
Precision Bio is taking a novel approach to gene editing to deploy a cure for patients with chronic hepatitis B.
Mark Forman, MD, PhD, on Genetic Testing and Further Research in Frontotemporal Dementia
The chief medical officer of Passage Bio discussed research needs in FTD.
CGTLive’s Weekly Rewind – September 2, 2022
Review top news and interview highlights from the week ending September 2, 2022.
FDA Recognizes CRISPR Therapy for Treatment of Hereditary Angioedema
The in vivo genome-editing therapy is the first gene therapy to be evaluated in a clinical trial for preventing HAE attacks.
Regen Developing Novel CAR Approach for Solid Tumors
The company has filed a patent for the new approach, which is designed to combat T-cell exhaustion.
Phase 3 Heart Failure Cell Therapy Trial Receives Go-Ahead from DSMB
The risk-benefit assessment determined that there are no significant safety concerns.
Developing a Safer Allogeneic Cell Therapy for Ovarian Cancer
Michael Heffernan, chairman and chief executive officer, Avenge Bio, discussed AVB-001's lower systemic toxicity profile seen in preclinical studies.
Cardiovascular Mesenchymal Cell Therapy Fast Tracked, Soon to Be Patented
Lomecel-B is being evaluated in the phase 2 ELPISII study in children with hypoplastic left heart syndrome.
Around the Helix: Cell and Gene Therapy Company Updates – August 31, 2022
Sangamo’s Gene Therapy Promising for Fabry Disease
Updated data from the STAAR clinical trial were presented at the SSIEM Annual Meeting.
New Imaging Approach Elucidates Vision Restoration After Gene Therapy in Achromatopsia
Researchers used novel functional MRI approaches to better measure the effects of gene therapy in children in current clinical trials.
Alexis Thompson, MD, MPH, on the Impact of Beti-Cel's Approval in Beta Thalassemia
The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed the collaboration and research that went into developing beti-cel.
CARsgen Advances Autologous CAR T Therapies in Multiple Myeloma, Advanced Gastric Cancer
The company has initiated the world’s first confirmatory phase 2 trial of a CAR T-cell therapy in solid tumors.
Mark Forman, MD, PhD, on ICM Administration of Gene Therapy in Frontotemporal Dementia
The chief medical officer of Passage Bio discussed the phase 1/2 upliFT-D study of PBFT02.
Developing a More Precise Allogeneic Cell Therapy for Ovarian Cancer
Michael Heffernan, chairman and chief executive officer, Avenge Bio, discussed the IND clearance of AVB-001 and the soon-to-initiate phase 1 trial.
Iovance Starts BLA Submission of Lifileucel for Advanced Melanoma
The tumor-infiltrating lymphocyte therapy has shown efficacy in the phase 2 C-144-01 trial.
CGTLive’s Weekly Rewind – August 26, 2022
Review top news and interview highlights from the week ending August 26, 2022.
Irene Agodoa, MD, on Addressing GVHD Risks in Treating Hematologic Malignancies
The senior vice president and head of medical affairs at Orca Bio discussed how Orca-T has been developed to lower the risk of graft-versus-host disease.
Allogeneic CAR-iNK Therapy to be Evaluated in B-Cell Malignancies
Century Therapeutics will initiate the phase 1 ELiPSE-1 study imminently.
Roctavian Approved for Hemophilia A in the EU
BioMarin plans to resubmit the biologics license application for val-rox to the FDA by the end of September 2022.
FDA Recognizes Ornithine Transcarbamylase Deficiency Gene Editing Therapy
iECURE and the University of Pennsylvania Gene Therapy Program have collaborated to develop the GTP-506 program.
Around the Helix: Cell and Gene Therapy Company Updates – August 24, 2022
Alexis Thompson, MD, MPH, on Qualifying Treatment Centers for Beti-Cel in Thalassemia
The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed requirements for administering beti-cel.
