Arshad Khanani, MD, on Reducing Treatment Burden in Wet AMD With Gene Therapy
The director of clinical research at Sierra Eye Associates discussed unmet needs in patients with wet AMD.
Around the Helix: Cell and Gene Therapy Company Updates – November 30, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
GM1 Gangliosidosis Gene Therapy Trial Safe to Continue
Lysogene’s MPS IIIA gene therapy program recently failed its primary endpoint in the phase 2/3 AAVANCE trial.
Etranacogene Dezaparvovec Approval May Pave Way for Val-Rox in Hemophilia A
The FDA has decided not to hold an advisory committee meeting for Roctavian after all.
FDA Recognizes Allogeneic CAR T Therapy for Lymphomas
CB-010 most recently demonstrated a 100% complete response rate in data presented at the EHA 2022 congress.
Base-Editing HSC Therapy Enrolls First Patient With Sickle Cell Disease
BEAM-101 is edited with a next-generation CRIPSR product to mimic single nucleotide polymorphisms that enable persistence of fetal hemoglobin.
ARV Cardiomyopathy Gene Therapy Gets Orphan Drug Designation
In preclinical research, TN-401 was shown to significantly increase the lifespan of PKP2-knock-out mice.
Sarepta’s DMD Gene Therapy Accepted for Priority Review
SRP-9001's PDUFA date is set for May 23, 2023.
CGTLive’s Weekly Rewind – November 25, 2022
Review top news and interview highlights from the week ending November 25, 2022.
NRTX-1001 Reduces Mesial Temporal Lobe Epilepsy Seizures in 2 Patients
The first participant has been followed-up for 3 months so far after treatment while the second is only 1 week post-treatment.
Around the Helix: Cell and Gene Therapy Company Updates – November 23, 2022
Etranacogene Dezaparvovec, Marketed as Hemgenix, Is First Gene Therapy Approved for Hemophilia B
A marketing authorization application for etranacogene dezaparvovec is currently under review by the EMA.
Legend Biotech Brings SCLC CAR T to Trials
The IND clearance comes after Legend terminated another clinical trial in T-cell lymphoma in July 2022.
Targeting Hematological Malignancies With Autologous and Allogeneic Cell Therapies
Thomas Willemsen, president and chief executive officer, Tessa Therapeutics, discussed the company’s technologies and 2023 milestones.
CaMMouflage Study to Evaluate Allogeneic Anti-BCMA CAR T Therapy in Multiple Myeloma
The phase 1 clinical trial plans to initiate enrollment in early 2023.
Gene Therapy to be Assessed in Heart Failure With Preserved Ejection Fraction Trial
SRD-001 is designed to increase expression and functional activity of SERCA2a, down-regulation of which is associated with all forms of heart failure.
New Afami-Cel Data to Support BLA Submission in Synovial Sarcoma
Updated data from the SPEARHEAD-1 study were presented at the 2022 CTOS Annual Meeting.
Lifileucel BLA Completion Pushed Back to Q1 2023
The FDA has requested supplemental validation information and comparability data for the tumor-infiltrating therapy.
CGTLive’s Weekly Rewind – November 18, 2022
Review top news and interview highlights from the week ending November 18, 2022.
Terese Hammond, MD, on Investigating iNKT Cells in COVID-Associated ARDS
The director of the CCU/ICU at Saint John’s Health Center discussed unmet respiratory treatment needs in the beginning of the COVID-19 pandemic.
Leber Congenital Amaurosis CRISPR Therapy Paused for Disappointing Efficacy
Editas has paused enrollment in the BRILLIANCE trial after efficacy was only seen in a very small subset of patients.
Combination Adenovirus Therapy Shows Survival Gains in Newly Diagnosed High-Grade Glioma
CAN-2409 transduces tumor cells with the thymidine kinase gene, sensitizes these cells to valacyclovir, and stimulates patients’ immune response.
Around the Helix: Cell and Gene Therapy Company Updates – November 16, 2022
CT-0508 Demonstrates Cytotoxicity in Solid Tumors
Updated data from a first-in-human trial were presented at the SITC 2022 meeting.
Omid Hamid, MD, on Improving Duration of Response in Refractory Melanoma With Lifileucel
The chief of research and immunotherapy at Cedars-Sinai The Angeles Clinic and Research Institute discussed updated data from the phase 2 C-144-01 study of the lifileucel TIL therapy.
Gene Therapy Restores Aflibercept Expression in Wet AMD
4D Molecular Therapeutics plans to initiate enrollment in the phase 2 randomized portion of the study in the first quarter of 2023.
Lupus Nephritis CAR-T Therapy Cleared for Phase 1/2 Clinical Trial
KYV-101 is an autologous version of a CD19-directed fully human CAR-T construct.
NTLA-2002 Reduces Hereditary Angioedema Attacks and Plasma Kallikrein
The first 3 patients dosed have been attack-free for up to 10.5 months.
New Research Identifies Safe Harbors for Gene Editing Therapies
The Genomics and Epigenetic Guided Safe Harbor mapper will aid in the future design of gene-editing therapies.
HLA LOH-Targeting Tmod Cell Therapies Demonstrate Selective Killing of Tumor Cells in Mice
A2B530 and A2B694 target CEA and MSLN cells that have HLA loss of heterozygosity.
