Victoria Johnson

Steven Pipe, MD, CS Mott Children’s Hospital, discussed the latest follow-up data from the HOPE-B study of the approved therapy, Hemgenix.

A phase 1 clinical trial of EXG102-031 is expected to initiate in the first quarter of 2023.

Brian Koffman, MDCM, DCFP, FCFP, DABFP, MSEd, Co-Founder, Executive Vice President, and Chief Medical Officer, CLL Society, discussed the role the society plays in the field.

Athersys has made progress in enrollment with its MATRICS-1 and MASTERS-2 clinical trials.

huMNC2-CAR22 is designed to avoid off-tissue expression and reduce T-cell exhaustion.

The new analysis data was requested by the FDA ahead of the therapy’s March 31, 2023 PDUFA date.

Phase 1/2 studies evaluated PDA001 in 50 participants with CD.

CKO804 is being evaluated as an add-on therapy to ruxolitinib in patients whose disease had a suboptimal response to ruxolitinib alone.

The company is kicking off 2023 by announcing planned advancements for several gene therapies in its pipeline.

The BALLI-01 study, initiated in 2019, has now dosed its first patient with an in-house manufactured CAR T-cell therapy candidate.

Both REGENXBIO and Rocket Pharmaceuticals have announced trial updates in their gene therapy programs for rare diseases.

Invectys and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience, announced the therapy and collaboration in June 2022.

Patients had an ORR of 83.8% with a median DOR of 15.7 months in data presented at the ASH 2022 meeting.

With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.

Half of wounds treated with the gene therapy had complete healing at both months 3 and 6 compared to 7% of placebo-treated wounds.

New data from a phase 1/2 trial of BMS-986393 were presented at the 64th Annual ASH Meeting.

Annualized bleed rate and FVIII infusion rates significantly decreased after infusion with the gene therapy.

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

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.

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.

Patients with non-Hodgkin lymphoma in Nkarta's phase 1 study had a 75% complete response rate.

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.

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.

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