Victoria Johnson

Other of the company’s RNAi therapies have recently shown promising data in hyperlipidemia and asthma.

Nine responses were ongoing as of the March 2023 cutoff date.

Of 300 screened participants, 68 have been matched by target antigen and HLA expression to a TCR-T in the ImmunoBank.

PFS jumped from under 3 months to over 14 when participants received CB-010 from a donor with at least 4 matched HLA alleles.

Current handling guidelines do not align between the laboratory and clinical settings.

No serious adverse events or significant retinal atrophy occurred.

An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.

Transient responses in 2 of 3 participants may be due to limited persistence of CARv3-TEAM-E T-cells in the weeks after infusion.

Fifty percent of patients had stable disease or better, with 2 complete responses and 2 partial responses.

B-VEC is approved in a gel formulation under the name Vyjuvek for skin wounds in dystrophic epidermolysis bullosa.

Ocugen’s gene agnostic modifier gene therapies include OCU400 and OCU410.

Tab-Cel is approved under the name Ebvallo in Europe, Switzerland, and United Kingdom.

Excision is evaluating a higher dose in a second cohort as well as exploring alternative, potentially redosable, delivery methods.

Most participants had improved or stable cognitive function in a proof-of-concept study.

Jonathan W. Weinsaft, MD, chief of cardiology and professor of medicine at Weill Cornell Medical College, discussed research the center is doing in the space.

Oncological trials of biologics also face particularly higher rates of clinical holds.

Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.

The CAR T-cell therapy was also recently approved for treating chronic lymphocytic leukemia or small lymphocytic lymphoma.

Eladocagene exuparvovec is approved in the United Kingdom and European Union under the name Upstaza.

High dose MCO-010 yielded statistically significant improvements in BCVA and MLSDT at week 52.

The trial is set to dose its second cohort following a positive DSMB recommendation in March.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

All 139 tumors screened had enhanced expression of CCNG1, a seldom-recorded gene on NGS.

The gene therapy is up for priority review with a PDUFA date of June 21, 2024.

The DSMB recommended proceeding to dose the highest dose cohort in the phase 1/2 PRODYGY study.

MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.

In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial.

KL003 favorably compares to platelet and neutrophil recovery rates of approved gene therapies.

Lovo-cel was approved as Lyfgenia in December 2023.

The ODAC unanimously voted in favor of the use of minimal residual disease negativity.