Noah Stansfield

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.

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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.

The announcement comes after positive top-line results in the ongoing KarMMa-3 study.

Improved functional activity compared to ROR-1-targeted CAR-T therapies without the 2 forms of reprogramming was demonstrated in preclinical studies.

Of the 8 treated patients who were evaluable, 6 patients had stable disease at Day 28.

Among the 31 patients evaluable for efficacy analysis, ORR was 100%.

SLN124 was well-tolerated in a previous clinical trial involving healthy volunteers.

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