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Intellia's CRISPR Gene Therapy Shows Potential as Functional Cure for Hereditary Angioedema in Long-Term Study

a year ago

Intellia Therapeutics' NTLA-2002, an in vivo CRISPR-based gene editing therapy, demonstrated a 98% mean reduction in monthly hereditary angioedema attack rates across all patients, with follow-up extending beyond two years.
Eight of ten patients remained completely attack-free following the initial 16-week observation period, with the longest attack-free duration reaching over 26 months and continuing.
The single-dose treatment showed a favorable safety profile across all dose levels, with no serious adverse events reported, positioning NTLA-2002 as a potential functional cure for this rare genetic disease.

NCT05120830Active, Not RecruitingPhase 1

Intellia Therapeutics

Posted 12/10/2021

2 years ago

CRISPR/Cas9 gene editing technology is being integrated with CAR-T cell therapy to address key limitations including immune checkpoint inhibition, T cell exhaustion, and manufacturing challenges.
The technology enables precise knockout of immune checkpoint genes like PD-1 and CTLA-4, significantly enhancing CAR-T cell persistence and anti-tumor activity in preclinical studies.
CRISPR/Cas9 facilitates development of universal "off-the-shelf" CAR-T cells by eliminating TCR and HLA genes, potentially reducing manufacturing costs and treatment timelines from weeks to days.
Despite promising clinical trial results showing safety and feasibility, researchers continue addressing potential safety concerns including off-target effects and chromosomal instability.

2 years ago

Verve Therapeutics reported Phase 1 trial results showing their base editing gene therapy reduced LDL cholesterol by up to 55% in patients with familial hypercholesterolemia.
The treatment uses base editing to modify the PCSK9 gene in liver cells, potentially offering a one-time therapy to replace daily cholesterol medications.
While promising, the 10-patient trial raises safety concerns due to permanent DNA changes, requiring extensive long-term follow-up before broader approval.
Experts emphasize the need for larger studies and years of additional research before this personalized approach could become widely available.