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.
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.
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.
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.
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.
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.
Genprex Patents REQORSA Immunogene Therapy for NSCLC
The company recently announced a dose escalation in the ACCLAIM-1 study.
Homology Trades in Gene Delivery for Gene Editing to Treat Phenylketonuria
The pheEDIT clinical trial initiated in June 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.
ProQR Pivots From Ophthalmic to CNS, Liver RNA Therapies
The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.
Genprex to Evaluate Higher Dose of REQORSA in NSCLC
A safety review committee approved the dose-escalation after reviewing the first cohort of patients.
Bristol Myers to Fund Inflammatory Bowel Disease Treg Therapies
GentiBio will use their proprietary regulatory T cell platform to develop the engineered therapies against multiple targets.
Zolgensma Gene Therapy Linked to 2 Deaths in SMA Patients, Novartis Reports
The 2 deaths, due to acute liver failure, occurred in patients treated in Kazakhstan and Russia.
Frontotemporal Dementia Gene Therapy Trial Initiates Dosing
PBFT02, developed by Passage Bio, is delivered via intra cisterna magna administration to the brain.
CRISPR Therapeutic Ready to Dose in Duchenne Muscular Dystrophy
A single patient will be dosed with CRD-THM-001 in an upcoming trial.
NK Cell Therapy Trial Doses First Patient With Lymphoma
The FDA recently accepted the company’s BLA for omidubicel for priority review.
Ide-Cel Shows Benefit in Earlier Use in R/R Multiple Myeloma
A recent comparison of health-related quality of life scores also favored ide-cel over belantamab mafodotin treatment.
IND Cleared for Non-Hodgkin Lymphoma T-Cell Therapy
Marker will initiate a company-sponsored study after an initial trial at Baylor College of Medicine showed positive safety data.
Umbilical Cord Lining Stem Cell Therapy Trial Initiates for Autoimmune Disorders
The trial is being launched by the University of Florida Center for Regenerative Medicine in collaboration with RESTEM.
Allogeneic Cell Therapy Trial to Initiate in Ovarian Cancer
Avenge Bio previously presented positive preclinical data at the ASGCT 2022 meeting.
REGENXBIO's MPSII Gene Therapy Charts Course to Approval
The FDA will consider RGX-121's BLA in an accelerated approval pathway.
Diabetic Kidney Disease Cell Therapy Trial Completes Enrollment
Topline results are expected in the first quarter of 2023.
IND Cleared for Dual-Targeted CAR T Therapy for B-Cell Malignancies
UCART2022 is an allogeneic therapy that targets both CD20 and CD22.
Sarepta Planning BLA Submission of DMD Gene Therapy
The announcement follows positive data updates from the ENDEAVOR and SRP-9001-101 studies.
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