Victoria Johnson

The BCT-006-US trial is set to begin dosing this year.

Treated patients had a –0.2 mean change in cUHDRS compared with a –1.0 change in external control data.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

The Prescription Drug User Fee Act (PDUFA) target action date is August 4, 2024.

The trial's primary endpoint is improvement on Luminance Dependent Navigation Assessment (LDNA).

First-in-human data with low dose VRON-0200 were presented at the EASL 2024 congress.

The company has reported data in NMOSD, myasthenia gravis, and necrotizing myopathy over the last several months.

Patients in FUMANBA-2 received eque-cel after being ineligible for ASCT after 4 cycles of induction therapy.

CGTLive takes a look at the path gene therapies have blazed in leukodystrophies, the challenges they’ve faced, and the road they’ve paved.

Eight patients receiving either TSC-100 or TSC-101 have been disease-free for a median of 10 months of follow-up.

The FDA has requested limited additional CMC information.

Three participants with ATTR amyloidosis have received follow-on doses of NTLA-2001 with no serious complications for up to 3 years of follow-up.

All 3 participants who have follow-up of at least 1 year have met the endpoint so far.

The CLARA trial has also completed enrollment of participants with corneal edema secondary to corneal endothelial disease.

AMT-130 has shown some evidence of dose-dependent clinical benefits in treated study participants.

Fractyl Health is conducting IND-enabling studies for Rejuva.

KYV-101 racks up another potential indication in addition to its primary autoimmune investigations.

Data from up to 5 and 6 years of follow-up were presented at the 2024 EHA Congress.

An investigator noted challenging side effects related to immunosuppressant treatment.

Treated participants have achieved some mild improvements on MDS-UPDRS Part III at 18 months posttreatment.

Earlier and prophylactic interventions have enabled low rates and severity of AEs in recent years of administration.

Eleven of 12 efficacy-evaluable patients with SLE and LN have met the DORIS criteria for complete remission.

AOC 001 previously demonstrated benefit in patients in the phase 2 MARINA trial.

The FDA granted the designation after reviewing safety and efficacy data from phase 1 of the trial.

The phase 1/2 trial is proceeding to its phase 2 trial after safety was demonstrated in its first phase.

Candel also recently received orphan drug designation for CAN-3110 for treating recurrent, high-grade glioma.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Nine of 14 participants receiving EDIT-101 had meaningful improvements in at least 1 key functional outcome.

ADI-001 is also being evaluated in a phase 1 study in patients with B-cell non-Hodgkin lymphoma.

BSB-1001 targets HA-1 miHA and will be evaluated in the 1 phase 1/2a TCX-101 trial.