Victoria Johnson

The chief executive officer of Tessera Therapeutics discussed ongoing research into developing its platform for sickle cell disease, CAR T-cell therapy, and more.

CT-0525 is being evaluated in a phase 1 clinical trial currently enrolling participants.

The chief medical officer and head of translational research at Ring Therapeutics discussed research presented at ASGCT 2024.

Ractigen is also evaluating saRNA therapies for ALS and SMA.

The cofounder and chief science officer of Longeveron discussed updated data from the phase 2a CLEAR MIND study.

The European Commission granted conditional marketing authorization under the name Beqvez to the European Union.

There were no serious adverse events related to the cell therapy and no evidence of ARIA.

Omar Nadeem, MD, clinical director, Myeloma Cellular Therapies, Dana-Farber Cancer Institute, discussed GPRC5D-targeted CAR-T, BMS-986393.

The company acquired the program, along with Spark Therapeutics’ pipeline, in December 2019.

IASO also received IND clearance for a trial in myasthenia gravis in April 2024.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison discussed the state of cell and gene therapy development.

Most participants maintained only over a 5% FVIII level after at least 15 months of follow-up.

Both bluebird bio and Vertex Pharmaceuticals have received unfavorable opinions from the Office of the Inspector General on the matter.

After 3 tries, Mesoblast finally has a PDUFA date for its MSC therapy.

The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.

Of 724 patients treated at the center, the cumulative incidence of secondary hematologic malignancy at 3 years posttreatment was 6.5%.

The chief medical officer of Cartesian Therapeutics discussed data presented at ASGCT 2024 from a phase 2a study.

The full 52-week analysis of the phase 2 LUNA trial are expected in the first half of 2025.

Longeveron will present updated data from the phase 2a CLEAR MIND study at AAIC 2024 at the end of July.

The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.

The FDA announced its START Pilot Program in October 2023 with a similar goal.

Patient enrollment is anticipated to begin in the first quarter of 2025.

The senior researcher at Seattle Children’s discussed the development and validation of A2-CAR-CISC EngTreg cells.

Data from the high-dose cohort of a phase 1/2 clinical trial in South Korea will be shared in Fall 2024.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison shared insights from the 2024 ISCT meeting presidential plenary.

Despite the improvements, Lexeo was unable to discern a benefit on cardiovascular fitness and Peak VO2 from LX2006 with current measurements.

The medical director of clinical development at AskBio discussed setbacks in the phase 1 trial of AB-1002 gene therapy.

RG6501/OpRegen was well-tolerated, with no serious treatment-related AEs reported in follow-up data from a phase 1/2 trial.

1x1015 vg 4D-710 has been identified as the maximum tolerated dose in the AEROW trial.

GT Biopharma anticipates initiating the phase 1 clinical trial in the second half of 2024.