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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Achieving Deep Responses With CART-ddBCMA in Relapsed/Refractory Multiple Myeloma
Matthew J. Frigault, MD, discussed safety and efficacy findings on CART-ddBCMA.
Long-Term RELIANCE Trial Data Shows Durability With Relma-Cel in LBCL
At a median follow-up of 24 months, the best ORR achieved was 77.6% among 58 evaluable patients with large B-cell lymphoma treated with relma-cel.
Resurrecting ALS Mesenchymal Cell Therapy for Regulatory Approval
Merit Cudkowicz, MD, discussed corrected data from a phase 3 trial of NurOwn that BrainStorm believes supports a BLA submission.
Verismo Looks to Assess KIR-CAR Cell Therapy in Solid Tumors
If approved, the company will initiate the first-in-human STAR-101 phase 1 clinical trial.
Irene Agodoa, MD, on Assessing Orca-T for Safer Cell Therapy in Hematologic Malignancies
The senior vice president and head of medical affairs at Orca Bio discussed the initiation of the Precision-T phase 3 trial.
Dystrophic Epidermolysis Bullosa Gene Therapy BLA Accepted With Priority Review
The BLA was supported by data from 2 intrapatient, placebo-controlled clinical trials.
CGTLive’s Weekly Rewind – August 19, 2022
Review top news and interview highlights from the week ending August 19, 2022.
Genprex Patents REQORSA Immunogene Therapy for NSCLC
The company recently announced a dose escalation in the ACCLAIM-1 study.
Alexis Thompson, MD, MPH, on Beti-Cel's Approval in β-Thalassemia
The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed beti-cel's approval.
Homology Trades in Gene Delivery for Gene Editing to Treat Phenylketonuria
The pheEDIT clinical trial initiated in June 2022.
Around the Helix: Cell and Gene Therapy Company Updates – August 17, 2022
FDA Approves Beti-Cel Gene Therapy for β-Thalassemia
ZYNTEGLO is now the world's most expensive gene therapy at a $2.8 million price tag.
First Patient With Classical Hodgkin Lymphoma Dosed With CAR T/Nivolumab Combo
Tessa Therapeutics presented positive data from the phase 2 CHARIOT study of TT11 in December 2021.
Regen Files for Patent on CAR T-Cell Exhaustion Prevention Technology
Multiple cell and gene therapy companies have been granted patents for novel technology in 2022.
