Autolus Submits Obe-Cel BLA for Adult R/R B-ALL
The company also plans to submit an MAA to the EMA in the first half of 2024.
FDA to Investigate Risk of T-Cell Malignancy After CAR T-Cell Therapy
The FDA has received reports of multiple T-cell malignancies in patients after approved CAR-T treatment.
Second Gene Therapy for Epidermolysis Bullosa up for Priority Review
Pz-cel has a PDUFA date of May 25, 2024.
Gracell’s CAR T-Cell Therapy to be Evaluated in Lupus
GC012F recently yielded a 100% ORR in an investigator-initiated trial in patients with newly diagnosed multiple myeloma.
Enabling Wider Antigen Selection With Logic-Gated CAR T-Cell Therapy for Solid Tumors
Maria Pia Morelli, MD, PhD, assistant professor, MD Anderson Cancer Center, discussed her work on the EVEREST-1 trial of logic-gated CAR T-cell therapy currently being evaluated in colorectal and pancreatic cancers.
BrainStorm to Meet With FDA on Confirmatory Trial for NurOwn ALS Therapy
After a negative AdComm meeting and withdrawing its BLA, the company seeks guidance on a phase 3 trial.
Iovance’s NSCLC Trials Push Second TIL Therapy Closer to Submission
The PDUFA date for Iovance’s lead TIL lifileucel for melanoma was recently pushed back to February 24, 2024.
Patients With Congestive Heart Failure Show Clinically Meaningful Improvements After Gene Therapy
AskBio presented data from a phase 1/2 trial of AB-1002 gene therapy at the AHA Scientific Sessions 2023 meeting.
Sanfilippo Awareness Day: A Peek Into the CGT Pipeline
CGTLive takes a look at MPSIII therapies in the pipeline for World Sanfilippo Awareness Day on November 16.
Ocugen Doses First Patient With Stargardt Disease With Gene Modifier Therapy
The company most recently reported data from a trial in retinitis pigmentosa and Leber congenital amaurosis demonstrating OCU400’s gene-agnostic mechanism of action.
Single RNAi Therapy Injection Controls Mild-to-Moderate Hypertension for 6 Months
Data on zilebesiran from the KARDIA-2 trial, in combination with other hypertension medicines, are expected in 2024.
Kyverna’s KYV-101 CAR T-Cell Therapy Expands Autoimmune Investigations Into Myasthenia Gravis
The therapy is already being evaluated in patients with lupus nephritis and diffuse cutaneous systemic sclerosis.
CRISPR Editing Therapy Reduces LDL-C in Familial Hypercholesteremia in Small Trial
One serious AE of myocardial infarction may have been related to VERVE-101 therapy while other AEs of fatal cardiac arrest and tachycardia were determined to be unrelated to the therapy.
First Clinical Trial for NGLY1 Deficiency Coming to Evaluate New Gene Therapy
The trial will follow a phase 1/2/3 design and will evaluate GS-100 in pediatric patients with NGLY1 deficiency.
TCR-T Therapy Yields Some Responses in HBV-Related Hepatocellular Carcinoma
The median progression-free survival was 11 months and the SCG101 was well-tolerated.
Adzynma Engineered ERT Therapy Approved for Prophylactic, On-Demand cTTP Treatment
The therapy was previously granted priority review in May 2023.
Lynch Syndrome Neoantigen Vaccine is Well-Tolerated, Demonstrates Immunogenicity
Microsatellite instability (MSI) tumors are frequently found in people with Lynch syndrome, one of the most common hereditary colorectal cancers.
CRISPR CAR Therapy Shows 100% ORR in NHL in Chinese Trial
BRL Medicine is also enrolling a phase 1/2 multicenter trial evaluating BRL-201 at lower dose ranges.
T-Cell Engagers Show Potential in HPV16+ and WT1+ Cancers
Patients with HNSCC had a median overall survival (OS) of 20.8 months and a 12-month OS rate of 59%.
The Future of Dermatology? B-VEC's Impact in DEB 6 Months Post Approval
CGTLive spoke to experts in the field about their experiences and impressions of B-VEC almost half a year after its approval for dystrophic epidermolysis bullosa.
CLDN6-Targeted CAR Shows Some Signs of Activity in Solid Tumors
CARVac RNA vaccine may amplify the persistence of the BNT211 CAR T-cell therapy.
CRISPR Therapy Well Tolerated, Detectable in First 3 Participants With HIV
Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.
ELEVIDYS Study Fails Primary End Point in DMD, Secondary End Points Show Statistically Significant Benefit
Despite the announcement, CEO Doug Ingram shared that the company is seeking a wider approval for delandistrogene moxeparvovec-rokl regardless of age or ambulatory status.
Auditory Improvements Seen in First Child Treated With OTOF Hearing Loss Gene Therapy
Other efforts to develop a gene therapy for OTOF congenital hearing loss include programs from Sensorion and Eli Lilly.
Islet Cell Pouch Implantation Shows Promise in Type 1 Diabetes
One patient had their Cell Pouch removed after the donor islet cells were found to be contaminated with Candida albicans yeast.
Mustang Bio’s CAR/Oncolytic Virus Combo Cleared for Glioblastoma/Astrocytoma Trial
The combo, termed MB-109, consists of the MB-108 oncolytic virus and MB-101 CAR T-cell therapy.
Gaucher Disease Type 1 Gene Therapy Shows Promising Initial Data
The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.
Positive Preclinical Data Support FTD-GRN Gene Therapy Study Initiation
AviadoBio announced the initiation of the phase 1/2 ASPIRE-FTD clinical trial earlier in October.
REGENXBIO’s ABBV-RGX-314 Trials Aim to Reduce Treatment Burden in Inherited Retinal Diseases
The company expects that pivotal ATMOSPHERE and ASCENT trials will support a BLA filing in 2024 for wet AMD, while the AAVIATE trial evaluates the therapy in diabetic retinopathy.
bluebird's HGB-210 Trial Hopes to Support First SCD Gene Therapy Approval
Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.
2 Clarke Drive Cranbury, NJ 08512