Noah Stansfield

OCU400 utilizes Ocugen’s modifier gene therapy platform, which may allow the product to treat multiple retinal diseases.

Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.

Preclinical research showed the therapy was able to reduce cellular HIV infection by up to 99% in an in vitro model and more than 97% in a mouse model.

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

The therapy was previously granted orphan drug designation for both RP and Stargardt disease.

Nearly 20% of treated patients achieved at least 25% improvement in timed 25-foot walk speed and/or 9-Hole Peg test at 28 weeks.

ALLO-501A received RMAT designation from the FDA in June 2022.

The FDA recently cleared Freeline Therapeutics to begin treating patients in the United States.

Significant improvements were reported in full-field stimulus testing.

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

Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.

Among the 11 patients evaluated for efficacy, 9 patients (81.8%) had objective responses at 28 days.

A prior resubmission in June 2022 was delayed with requests for additional durability and safety data.

Of the 17 patients treated, 12 patients achieved a response, including 6 who achieved a complete response.

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

NR082 has been evaluated in 3 previous investigator-initiated trials involving 186 patients.

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

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

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

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

Interim data showed a 78% overall response rate and a 67% complete response rate.

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

The grant will specifically help support the trial’s treatment arm for patients with post-transplant acute myeloid leukemia with minimal residual disease.

Preliminary data from an ongoing clinical trial suggest that AVR-RD-04 has been well-tolerated.

In a mouse model of MPS II, IDS.ApoEII effected completely normalized brain pathology and behavior.

Adverum Biotechnologies’ multicenter, double-masked LUNA trial will enroll 72 patients aged 50 years or older.

A substantial benefit noted for EtranaDez and val-rox is that they are delivered as a single-dose.

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

In nonclinical studies, AK-OTOF was well-tolerated in both mice and non-human primates.

The disease models used also helped to establish the function of NPHP5 in cilia morphogenesis.