Victoria Johnson

The FDA has decided not to hold an advisory committee meeting for Roctavian after all.

CB-010 most recently demonstrated a 100% complete response rate in data presented at the EHA 2022 congress.

BEAM-101 is edited with a next-generation CRIPSR product to mimic single nucleotide polymorphisms that enable persistence of fetal hemoglobin.

SRP-9001's PDUFA date is set for May 23, 2023.

The first participant has been followed-up for 3 months so far after treatment while the second is only 1 week post-treatment.

A marketing authorization application for etranacogene dezaparvovec is currently under review by the EMA.

The IND clearance comes after Legend terminated another clinical trial in T-cell lymphoma in July 2022.

The phase 1 clinical trial plans to initiate enrollment in early 2023.

SRD-001 is designed to increase expression and functional activity of SERCA2a, down-regulation of which is associated with all forms of heart failure.

Updated data from the SPEARHEAD-1 study were presented at the 2022 CTOS Annual Meeting.

The FDA has requested supplemental validation information and comparability data for the tumor-infiltrating therapy.

Editas has paused enrollment in the BRILLIANCE trial after efficacy was only seen in a very small subset of patients.

CAN-2409 transduces tumor cells with the thymidine kinase gene, sensitizes these cells to valacyclovir, and stimulates patients’ immune response.

Updated data from a first-in-human trial were presented at the SITC 2022 meeting.

4D Molecular Therapeutics plans to initiate enrollment in the phase 2 randomized portion of the study in the first quarter of 2023.

The first 3 patients dosed have been attack-free for up to 10.5 months.

A2B530 and A2B694 target CEA and MSLN cells that have HLA loss of heterozygosity.

Data from the COBALT-RCC study in renal cell carcinoma were presented at SITC 2022.

Further analysis will be performed to elucidate which characteristics correlate with optimal cell therapy behavior.

The FDA action follows a slew of recent cell and gene therapy updates in the field of amyotrophic lateral sclerosis.

A final evidence report suggests a $1.9 million cap for val-rox and a $2.9 million cap for EtranaDez.

No treatment-related adverse events have been reported and Verve expects a letter detailing the hold within 30 days.

Abeona has announced topline results from its phase 3 VIITAL study of EB-101.

Updated data from the ALTITUDE trial were presented at the 55th ARS meeting in Pasadena, CA.

Data from the first 2 patients were presented at the IWWM 2022 meeting.

Neurotech has announced updated data from protocol A and B phase 3 studies.

The DSMB has recommended the trial to continue with additional risk mitigation procedures.

Cellular Biomedicine group developed the therapy after efficacy was seen with Iovance’s lifilecuel in solid tumor studies.

CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.

The trial expects to enroll its first patient in the first half of 2023.