Victoria Johnson

Participants treated both unilaterally or bilaterally reported clinically meaningful improvements on GRCQ and XQ scale scores.

The company announced that it had completed dosing in the first cohort of the GALILEO-1 trial of FLT201.

Surveyed clinicians and nurses from Rady Children's Hospital San Diego identified important factors and considerations needed in a potential SMD tool.

Bluebird's gene-edited cell therapy has a PDUFA date of December 20, 2023.

Mustang reported new data and responses from a multicenter phase 1 trial evaluating its CAR T-cell therapy in patients with NHL, FL, and WM.

Both Adaptimmune and Replay have placed their bets on targeting cancer testis antigens for synovial sarcoma and myxoid/round cell liposarcoma cell therapy.

The first participant was dosed with LBL-033 in April 2023 in Nanjing Lead Biolabs's ongoing trial in China.

The TIL therapy's PDUFA date has been pushed back to February 24, 2024.

At dose level 2, 3 of 4 participants had increases of β-Gal activity to normal levels for up to 12 months.

The phase 2 ALPHA2 trial of ALLO-501A should complete enrollment in 2024.

Four years after submitting its initial BLA for remestemcel-L for steroid-refractory acute graft versus host disease, Mesoblast has encountered another hurdle toward possible approval.

The gene-edited cell therapy OTL-200 was approved in Europe in 2020.

LEXEO also recently completed dosing in the first cohort of a trial for Friedreich’s ataxia cardiomyopathy.

The first-in-human MuST trial is being conducted in several centers in Germany.

With up to 20% of incidences of FTD driven by genetic mutations, multiple gene therapy companies have initiated clinical trials of AAV-mediated treatments for a patient population with little to no options.

The FDA cleared the IND for BRG01 in February 2023.

A DSMB futility analysis found that the trial was unlikely to meet its primary composite endpoint and trial enrollment has been paused.

The phase 1/2 trial evaluating EBT-101 dosed its first patient in September 2022.

Rocket Pharma is working to soon initiate a phase 1 trial of RP-A601.

Data on cohort 2 of the AEROW trial are expected in November 2023.

The updated report maintains exa-cel's C++ grade and lovo-cel's B+ grade.

The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.

The proof-of-concept trial was in healthy volunteers, and additional studies are being planned in patients with blood cancers undergoing hematopoietic stem cell transplants.

Invimestrocel is being investigated for multiple indications, including ischemic stroke.

Hemogenyx is currently remanufacturing the therapy without the identified splicing issue.

Wave Life Sciences is discontinuing WVE-004.

ACE1831 is Acepodia’s first γδ T-cell therapy to enter clinical trials.

Reductions in NfL were also associated with changes on ALSFRS-R scores.

A trial in China was initiated in December 2022 for patients with neovascular and wet age-related macular degeneration.

Data from patients treated in early access programs were presented at the 9th EAN Congress.