Canavan Gene Therapy Improves Developmental Measures Compared to Natural History
MSEL 5-mean domain, receptive language, and expressive language scores were significantly improved from baseline in treated participants.
Intellia’s CRISPR Therapy for ATTR-CM First to Enter Late-Stage Trials
Intellia Therapeutics plans to initiate the phase 3 trial of NTLA-2001 by the end of 2023.
NK Cell Therapy Shows Early Antitumor Response in B-NHL
The full data readout from the phase 1/2 clinical trial is expected in the first quarter of 2024.
TCR-T Therapy Cleared for Lung, Gastric Cancer Trial
Biosyngen is also evaluating the CAR T-cell therapy BRG01 in a phase 1/2 trial for EBV-positive relapsed/metastatic nasopharyngeal carcinoma.
Hold Lifted on ASO for Myotonic Dystrophy Type 1, Trial to Initiate in the US
The FDA previously placed the trial on clinical hold for undisclosed reasons.
BioCardia Targets Smaller Population of Heart Failure With Proposed Modified Study Protocol
The company aims to assess CardiAMP in patients with NT-proBNP over 500 pg/ml at baseline with a modified composite efficacy endpoint.
CGT Therapies More Likely to Gain Approval for Hematological Malignancies, Orphan Diseases
A NEWDIGS analysis compared clinical trial success rates from 1988 to 2020.
Antitumor Activity and Bone Regeneration: Tackling Multiple Angles of Osteosarcoma
Fiona Freeman, PhD, assistant professor, University College Dublin, discussed her research into microRNA-29b in suppressing tumor growth and promoting bone remodeling in mice models.
REGENXBIO’s DMD Gene Therapy Well-Tolerated, Efficacy Signals Promising
The AFFINITY trial is set to dose patients at dose level 2 by the end of 2023.
Gene Therapy Increases GCase Activity in Patients With Gaucher
The updated data are from the first 2 patients treated in the phase 1/2 GALILEO-1 trial of FLT201.
Patients With Type 1 Diabetes Independence Achieve Insulin Independence With Islet Cell Therapy
All patients showed improved glycemic control and a reduction in insulin independence.
2023 AcCELLerate Forum Encourages Discussion on Cell Therapy Landscape, Management, Future
The partnership between ASTCT, CIBMTR, and NMDP is a 2-day forum held October 2-3.
Rocket’s LAD-I Gene Therapy BLA Accepted for Priority Review
The therapy has a PDUFA date of March 31, 2024.
Epigenetic Therapy Shows Signs of Efficacy in MYC-Expressing Solid Tumors
The new data are from the phase 1/2 MYCHELANGELO trial of OTX-2002, an mRNA OEC therapy, in patients with hepatocellular carcinoma and other MYC-expressing tumors.
Dose-Limiting AAV Toxicity Responsible for Cure Rare Disease’s DMD Trial Death
Unforeseen complications may arise when treating older patients with gene therapy.
Axi-Cel Shows Efficacy in Patients With R/R LBCL Ineligible for ASCT
Around half of patients with R/R large B-cell lymphoma are considered ineligible for autologous stem cell transplant and are thus ineligible for the approved indication of Yescarta.
Abeona's RDEB Cell Therapy Up for Review
Abeona has submitted a BLA for its EB-101 cell therapy.
Patients Receiving Autologous Stem Cell Transplant After Chemical Burns Show Corneal Improvements
The phase 1/2 trial is opening its second phase of recruitment after being well-tolerated in the first 4 patients and demonstrating some vision improvements.
Optic Atrophy Gene Therapy Cleared for Trials in Australia
NFS-05 is an ophthalmic injection that uses an AAV vector to deliver the OPA1 gene to retinal ganglion cells.
Mesoblast Gears Up for Third Push for Remestemcel-L's Approval in GvHD
The FDA issued a CRL for the therapy, to be marketed as Ryoncil, in August 2023.
Research Identifies Cell Signatures of CAR T-Cells in Long Remission
Cells from patients with ALL treated in the CARPALL study were analyzed and sequenced.
First Patient Dosed With LCA5 Gene Therapy
Opus Genetics plans to add a pediatric cohort to the phase 1/2 trial once safety is established in adults.
Anixa Makes Progress in CER-T Trial for Ovarian Cancer
The trial has dosed its third patient, and, safety validation pending, plans to dose a second cohort beginning at the end of 2023.
Hemogenyx Clears a Path for AML CAR-T Therapy
The FDA has accepted the company’s plan to address its current IND clinical hold on HEMO-CAR-T.
Addition of Quizartinib FLT3 Inhibitor to Cell Transplant Improves Survival in AML
Patients were newly diagnosed and had acute myeloid leukemia positive for FLT3-iTD mutations.
First Patient Receives Gene Therapy for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Adstiladrin was approved in December 2022 and is now available for select patients under an Early Experience Program.
First Patient With MSLN-Solid Tumors Receives SynKIR-110 CAR T-Cell Therapy
SynKIR-110 is based off of the KIR-CAR platform developed at the University of Pennsylvania.
Multi-Base–Edited CAR T-Cell Therapy Trial Doses First Patient With R/R T-ALL/T-LL
Beam Therapeutics also expects to announce initial data from its BEACON trial in sickle cell in 2024.
RNAi Therapeutic Shows Dose-Dependent Reductions in Systolic Blood Pressure
Zilebesiran is being evaluated as a monotherapy in the KARDIA-1 study and as a combination therapy in KARDIA-2, which will report topline data in early 2024.
Nusinersen Show Benefits in Other SMA Populations
New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.
