News

SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.

A phase 1 and phase 1b study of CK0803 will soon be initiated following the FDA’s IND clearance.

CTX-130 is being investigated in phase 1 studies for relapsed/refractory T or B-cell malignancies and renal cell carcinoma.

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

Phil Cyr, Senior Vice President, Precision Value & Health, discussed challenges to tackle with CAR T-cell therapies.

Bluebird bio’s ZYNTEGLO was the first gene therapy to be approved in the space in August 2022.

Although efficacy and HGF expression were assessed as outcome measures, comparisons were not able to be made between placebo and Engensis groups.

The 3 patients demonstrated a mean significant improvement of up to 200% of baseline in motor unit potential.

Hong Ma, senior vice president, clinical development, cancer immunotherapy, CARSgen, discussed data from a phase 1 trial of CT041.

The 2-stage, multicenter clinical trial will recruit approximately 39 patients who have been diagnosed with LGMDR9.

The chief medical officer of Triumvira Immunologics discussed how TAC01-HER2 could address unmet needs in treating solid tumors.

Verismo plans to begin enrollment in the STAR-101 trial in the first quarter of 2023.

Elimination of the miR-17 binding site on PKD1 and PKD2 mRNA alleviated cyst growth in preclinical models.

The deputy director of the Masonic Cancer Center discussed trends of presentations from ESMO 2022.

The patient is expected to qualify for analytical treatment interruption of background anti-retroviral therapy.

Review top news and interview highlights from the week ending September 23, 2022.

The FDA previously lifted its clinical hold of the AFFINE study in May 2022.

The company previously presented positive preclinical data at the 2021 ASGCT meeting.

The professor of genetics at Federal University of Rio Grande do Sul discussed updated data from the CAMPSIITE trial.

Eleven patients in the LUMMICAR-2 trial were evaluable for preliminary efficacy analyses.

The chief medical officer of Triumvira Immunologics discussed updates from the TACTIC-2 study presented at ESMO Congress 2022.

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

The FDA has cleared the investigational new drug application for AB-201.

The deputy director of the Masonic Cancer Center discussed the preclinical findings of his study on a TriKE engager and NK cells.

Seven of 10 evaluable patients achieved minimal residual disease negativity after cilta-cel treatment.

Scientist have proposed several methods for converting stem cells into RPE, but there is still a gap in our knowledge of how cells respond to these stimuli over time.

The approval follows unanimous support of eli-cel's benefits in CALD from an FDA AdComm meeting and an approval for beti-cel in beta-thalassemia.

Administration of NTLA-2001 led to rapid and deep reductions in serum TTR by day 28.