News

A DCE analysis weighed gender-, age-, and disease-severity-specific annual costs as opposed to traditional cost-effectiveness models.

Additional data on 2 patients who developed persistent anemia suggests a genetic cause may be behind the serious adverse event.

Achilles Therapeutics presented updated data from CHIRON and THETIS at the 2022 ESMO immuno-oncology congress.

AVROBIO plans to initiate a global phase 2/3 trial of AVR-RD-02 in GD3 in the second half of 2023.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The allogeneic mesenchymal stem cell therapy trial will enroll patients ineligible for treatment with the autologous CardiAMP.

The first 2 participants dosed are free of vaso-occlusive events as of 5 and 1.5 months of follow-up after treatment with EDIT-301.

Ivan Horak, MD, discussed Tessa’s cell therapy platforms and the ACTION clinical trial.

Updated data were presented at the AES annual meeting in Nashville.

The associate professor from the Medical College of Wisconsin discussed how to address unmet needs with CAR T-cell therapies.

Initial safety data from the PRODYGY study are expected in 2023.

Beam Therapeutics provided data on genomic rearrangement, off-target edits and from a cytokine independent growth assay to the FDA in November 2022.

Review top news and interview highlights from the week ending December 2, 2022.

Updated data from 3 of the company’s programs were presented at an R&D showcase.

OPGx-001 is the first of Opus Genetics’ gene therapy candidates for treating LCA to enter clinical trials.

Sensorion indicated it is still on track to submit a clinical trial application in the first half of next year.

The director of clinical research at Sierra Eye Associates discussed unmet needs in patients with wet AMD.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Lysogene’s MPS IIIA gene therapy program recently failed its primary endpoint in the phase 2/3 AAVANCE trial.

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.

In preclinical research, TN-401 was shown to significantly increase the lifespan of PKP2-knock-out mice.

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

Review top news and interview highlights from the week ending November 25, 2022.

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

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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