Gene Therapy

ORLANDO — LentiGlobin gene therapy led to substantial reductions of sickle hemoglobin and considerable improvement in key markers of hemolysis, according to updated results from the phase 1/phase 2 HGB-206 study presented at TCT | Transplantation & Cellular Therapy Meetings.

Enrollment is complete in the two highest dose groups in a phase 1/2 clinical trial evaluating an adeno-associated virus-based gene therapy for the treatment of X-linked retinitis pigmentosa, according to a press release from Applied Genetic Technologies Corporation.

Four of six patients with neovascular age-related macular degeneration treated with a threefold lower dose of ADVM-022 in the OPTIC phase 1 trial did not require a rescue injection 24 weeks after treatment and demonstrated an efficacy signal with evidence of dose response.

The Bascom Palmer Angiogenesis meeting took place on Feb. 8, covering disease pathogenesis and emerging treatment options for various retinal conditions. The presentations highlighted how some of the newest imaging modalities are helping us understand the natural course of disease and new mechanisms of action being evaluated as alternatives to our current treatments. Below is just a mere highlight of many excellent presentations at the meeting.

Researchers found that cannabinoids did not appear to reduce cancer-related pain in adults. It was the week’s top story in hematology/oncology.

In 1984, a teenage boy was lying in his hospital bed, his body ravaged by acute graft-versus host disease.

Actinium Pharmaceuticals has forged an agreement with University of California, Davis for the use of its proprietary CD45 antibody radiation-conjugate, apamistamab-I-131, in the institution’s ongoing phase 1/phase 2 clinical trial of stem cell gene therapy for patients with HIV-related lymphoma.

KOLOA, Hawaii —Voretigene neparvovec benefits patients with Leber congenital amaurosis and may help advance future gene therapies, according to a speaker at Retina 2020.

KOLOA, Hawaii — An ophthalmologist whose life’s work has led to advancements in the field of genetic eye disease was honored here at Hawaiian Eye 2020.

Sickle cell disease, the most common inherited red blood cell disorder in the United States, is associated with debilitating physical symptoms, including acute pain crises, joint and organ damage, diminished cognitive function and decreased life expectancy.