Gene Therapy

Also known as Sanfilippo syndrome, this rare genetic syndrome has no known cures and what current treatment options are available are expensive and limited in supply.

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185). It was the second approval from FDA for a gene therapy for sickle cell disease.

Among 10 patients evaluated, compared with their baseline at 1 week after receiving their final dose in the trial, 30% demonstrated clinical improvement on the Alzheimer’s disease composite score.

Education, psychological support, and implementation guidance are the top unmet needs identified by investigators from the United Kingdom concerning gene therapy use for hemophilia.

Over a 52-week treatment period, the gene therapy demonstrated robust evidence for a clinically meaningful benefit, including across several prespecified functional secondary end points vs placebo.

The FDA approved delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics) in June to treat Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 who have a confirmed DMD gene mutation.

Investigators propose potential payment models for gene therapies that consider equitable patient access and payer reimbursement.

A child with profound genetic hearing loss was dosed with the investigational otoferlin gene therapy (DB-OTO), becoming the first patient dosed in a phase 1/2 trial evaluating the treatment, and experienced improved auditory response at week 6 with no concerning safety signals.

Sickle cell disease (SCD) has known complications that include organ damage and failure, rhabdomyolysis, glaucoma, splenic sequestration, and vaso-occlusive crises. At present, the only potential cure is a bone marrow transplant, but it can be difficult to find a donor and there is a high rejection risk.

The location of integration of a gene therapy has been crucial for the safety and efficacy of the treatment to cure infants with X-linked severe combined immunodeficiency.

In this case study, a patient who had Duchenne muscular dystrophy (DMD) and received high-dose transgene therapy to upregulate cortical dystrophin subsequently developed acute respiratory distress syndrome and died.

Both the second and first patients dosed in the study received the lower of the 2 doses of the gene therapy that Taysha Gene Therapies is evaluating in the phase 1/2 REVEAL trial.

Sarah Boyce, president and CEO at Avidity Biosciences, discusses her leading role at the company, as well as antibody oligonucleotide conjugate drug, AOC 1044, currently in development for Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping (DMD44).

The program will initially allow 6 sponsors of clinical trials to participate and will provide sponsors with the opportunity for frequent advice and regular communication regarding clinical trial design and other development issues.

The researchers focused on coverage trends across an array of disease states, including lymphoma, vision loss, and spinal muscular atrophy, finding discrepancies in coverage across 16 states and Medicaid Care Organizations.

The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene.

Many gene therapies promise life-changing effects, but without long-term data it remains to be seen how long the benefits last.

Experts evaluated the future of cell and gene therapies in the United States and offered insights on potential value-based payment models for these treatments.

With more than 3000 gene therapies in development, payers will have to grapple with the challenges of paying for these innovative but expensive therapies.