Gene Therapy

Gene therapies can be life-changing for people, but the high cost plus the burden of treatment remain barriers to access and utilization, explained Kevin Niehoff, PharmD, BCMAS, of IPD Analytics.

Investigators said patients experienced adverse effects in line with those expected for myeloablative conditioning with busulfan following treatment with betibeglogene autotemcel (beti-cel; Zynteglo; bluebird bio).

Further research is needed to determine how once-weekly viltolarsen can slow disease progression and preserve motor function in boys and young men with Duchenne muscular dystrophy (DMD).

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with the developer citing safety concerns, FDA feedback, and an evolving therapeutic landscape.

The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase (AADC) deficiency.

The gene therapy improved annualized bleed rates, though a handful of study participants resumed factor IX prophylaxis.

New legislation would clarify rules around value-based contracts in Medicaid, and a new model is expanding access to gene therapies, explained Adam Colborn, JD, of AMCP.

The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.

Investigators said the unique features of chimeric antigen receptor (CAR) T-cell therapy may warrant novel approaches to cost-effectiveness analysis.

A new study reviews how various routes of adeno-associated virus (AAV) gene therapy administration influence local and peripheral immune responses, with a focus on those observed in clinical trials targeting the central nervous system (CNS).

Three patients with RPE65-related retinopathy showed improved function in both eyes following gene augmentation therapy treatment with voretigene neparvovec.

Insulin resistance plays a role in many diseases. The new research provides insights into why insulin resistance appears to be associated with neurodegenerative disorders.

Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect profile that is consistent with high-dose steroid use in other conditions, according to a meta-study that analyzed immunosuppressive protocols.

A new proof-of-concept method reliably engineered a hotspot mutation of SF3B1, a gene-splicing gene, into diverse cancer cell lines, outperforming other contemporary editing approaches.

Early data suggest chimeric antigen receptor (CAR) T-cell therapy can achieve meaningful long-term results.

A case report highlights a rare instance of parkinsonism following CAR T-cell therapy for multiple myeloma.

Several years of preliminary research results suggest chimeric antigen receptor (CAR) T-cell therapy could benefit pediatric patients with refractory systemic lupus erythematosus (SLE).

The gene therapy landscape has changed dramatically in the past 5 years, and investigators wanted to capture and analyze the details of current and upcoming trials, along with their potential impact on patients.

Researchers introduce a chimeric antigen receptor–enhancer (CAR-E) therapeutic platform that significantly extends the effectiveness and memory of CAR T cells, potentially preventing relapse in patients who have cancer.

Experts argue for new mechanisms to fund research in oft-ignored rare diseases.

An exon-skipping technique using dual single-guide RNA/Cas9 ribonucleoproteins targeted at 3 novel COL7A1 exons with pathogenic heterozygous mutations achieved exon deletion rates of up to 95%.

An innovative CRISPR-Cas12f-based activator system may hold significant potential in the future of gene therapy and biological research, suggest researchers of a new study.

The gene therapy giroctocogene fitelparvovec demonstrated superiority compared with the standard of care, routine prophylaxis, in hemophilia A treatment.

Beremagene geperpavec-svdt (B-VEC) entered the market in 2023 as the first approved corrective treatment for dystrophic epidermolysis bullosa (DEB), a rare genetic disease affecting the skin and nails that is caused by mutations in the COL7A1 gene.

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as the challenges brought by manufacturing complexities and the need to address accessibility to treatment.

The therapy led to increased frataxin levels and decreased left ventricular mass in patients with Friedreich ataxia (FA) cardiomyopathy, the authors said.

The biomarker, G-protein-coupled receptor 176 (GPR176), may be a driver in the progression of ovarian cancer and potential target for gene therapy, suggested researchers of a new study.

The interim results of a single-arm trial in children with autosomal recessive deafness 9 (DFNB9) show the benefits of binaural administration of gene therapy: better hearing with no serious adverse effects.

An adeno-associated virus (AAV) gene therapy for hereditary spastic paraplegia type 50 (SPG50)—funded by a young patient’s family—gives hope not only to a 4-year-old, but to researchers working on similar projects for other rare diseases.

Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein.