SPK-8011 Yields Durable FVIII Expression in Hemophilia A
Annualized bleed rate and FVIII infusion rates significantly decreased after infusion with the gene therapy.
Gene Therapy for Sickle Cell Disease Cost Effective in Reducing Health Disparities
A DCE analysis weighed gender-, age-, and disease-severity-specific annual costs as opposed to traditional cost-effectiveness models.
Neoantigen Reactive T Cell Therapy Shows Early Signs of Efficacy in NSCLC and Melanoma
Achilles Therapeutics presented updated data from CHIRON and THETIS at the 2022 ESMO immuno-oncology congress.
Stem Cell Gene Therapy Show Clinical Improvements in Gaucher Type 1 and 3
AVROBIO plans to initiate a global phase 2/3 trial of AVR-RD-02 in GD3 in the second half of 2023.
BioCardia Follows up CardiAMP With CardiALLO Clearance for Heart Failure
The allogeneic mesenchymal stem cell therapy trial will enroll patients ineligible for treatment with the autologous CardiAMP.
Genome-Edited Cell Therapy Reduces VOEs in Sickle Cell Disease
The first 2 participants dosed are free of vaso-occlusive events as of 5 and 1.5 months of follow-up after treatment with EDIT-301.
Leveraging Cell Therapy Advances for Treating Hodgkin Lymphoma, Other Cancers
Ivan Horak, MD, discussed Tessa’s cell therapy platforms and the ACTION clinical trial.
Neural Cell Therapy Reduce Seizures in MTLE
Updated data were presented at the AES annual meeting in Nashville.
Nkarta's Allogeneic CAR T Shows Clinical Responses in Non-Hodgkin Lymphoma
Patients with non-Hodgkin lymphoma in Nkarta's phase 1 study had a 75% complete response rate.
Retinitis Pigmentosa Gene Therapy Cleared for Trials
Initial safety data from the PRODYGY study are expected in 2023.
Multiplex-Base Edited CAR T Therapy Cleared for Trial in T-ALL After Hold
Beam Therapeutics provided data on genomic rearrangement, off-target edits and from a cytokine independent growth assay to the FDA in November 2022.
Allogene’s CAR T Therapies Show Antitumor Activity in Solid and Liquid Tumors
Updated data from 3 of the company’s programs were presented at an R&D showcase.
Leber Congenital Amaurosis 5 Gene Therapy Cleared for Trials
OPGx-001 is the first of Opus Genetics’ gene therapy candidates for treating LCA to enter clinical trials.
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.
Hemgenix 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.
Sarepta’s DMD Gene Therapy Accepted for Priority Review
SRP-9001's PDUFA date is set for May 23, 2023.
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.
Hemgenix 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.
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.
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.
CT-0508 Demonstrates Cytotoxicity in Solid Tumors
Updated data from a first-in-human trial were presented at the SITC 2022 meeting.
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.
NTLA-2002 Reduces Hereditary Angioedema Attacks and Plasma Kallikrein
The first 3 patients dosed have been attack-free for up to 10.5 months.
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