Victoria Johnson

The PDUFA date for Iovance’s lead TIL lifileucel for melanoma was recently pushed back to February 24, 2024.

AskBio presented data from a phase 1/2 trial of AB-1002 gene therapy at the AHA Scientific Sessions 2023 meeting.

CGTLive takes a look at MPSIII therapies in the pipeline for World Sanfilippo Awareness Day on November 16.

The company most recently reported data from a trial in retinitis pigmentosa and Leber congenital amaurosis demonstrating OCU400’s gene-agnostic mechanism of action.

Data on zilebesiran from the KARDIA-2 trial, in combination with other hypertension medicines, are expected in 2024.

The therapy is already being evaluated in patients with lupus nephritis and diffuse cutaneous systemic sclerosis.

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.

The trial will follow a phase 1/2/3 design and will evaluate GS-100 in pediatric patients with NGLY1 deficiency.

The median progression-free survival was 11 months and the SCG101 was well-tolerated.

The therapy was previously granted priority review in May 2023.

Microsatellite instability (MSI) tumors are frequently found in people with Lynch syndrome, one of the most common hereditary colorectal cancers.

BRL Medicine is also enrolling a phase 1/2 multicenter trial evaluating BRL-201 at lower dose ranges.

Patients with HNSCC had a median overall survival (OS) of 20.8 months and a 12-month OS rate of 59%.

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.

CARVac RNA vaccine may amplify the persistence of the BNT211 CAR T-cell therapy.

Excision plans to escalate the trial of EBT-101 to the second dose level in the fourth quarter of 2023.

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.

Other efforts to develop a gene therapy for OTOF congenital hearing loss include programs from Sensorion and Eli Lilly.

One patient had their Cell Pouch removed after the donor islet cells were found to be contaminated with Candida albicans yeast.

The combo, termed MB-109, consists of the MB-108 oncolytic virus and MB-101 CAR T-cell therapy.

The therapy has been well-tolerated in the first 3 participants which also showed GCase expression in the plasma.

AviadoBio announced the initiation of the phase 1/2 ASPIRE-FTD clinical trial earlier in October.

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.

Lovo-cel is currently being evaluated in the phase 3 HGB-210 study and has a PDUFA date of December 20, 2023.

MSEL 5-mean domain, receptive language, and expressive language scores were significantly improved from baseline in treated participants.

Intellia Therapeutics plans to initiate the phase 3 trial of NTLA-2001 by the end of 2023.

The full data readout from the phase 1/2 clinical trial is expected in the first quarter of 2024.

Biosyngen is also evaluating the CAR T-cell therapy BRG01 in a phase 1/2 trial for EBV-positive relapsed/metastatic nasopharyngeal carcinoma.

The FDA previously placed the trial on clinical hold for undisclosed reasons.

The company aims to assess CardiAMP in patients with NT-proBNP over 500 pg/ml at baseline with a modified composite efficacy endpoint.