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.
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.