Gene Therapy

Early research shows a one-time gene therapy has potential for addressing both the liver and lung disease that patients with alpha-1 antitrypsin deficiency (AATD) face.

Researchers have suggested that uniform evidence standards and reporting requirements could help speed patient access to cell and gene therapies and help ensure consistent safety and efficacy evaluations.

Solid Biosciences’ gene therapy uses a shortened version of the dystrophin protein, which is engineered to mimic the key functions of full-length dystrophin and allows it be delivered more effectively to muscle cells.

A mouse study at Memorial Sloan Kettering Cancer Center has found that adding Nef, a protein from HIV, allows donor CAR T cells to avoid detection by the immune system while still attacking tumors.

The majority are in the very early preclinical stage of development, but applications for regulatory approval have been submitted for 11 gene therapies worldwide, and 35 are in phase 3 trials, according to report delineating the gene therapy development landscape.

Here are a few gene and cell therapy clinical trials with expected data readouts in 2025 that are worth keeping an eye on.

President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program that helps state Medicaid programs pay for expensive cell and gene therapy.

Cardiomyopathies currently have no cures, but many of these disorders are prime candidates for gene therapies.

Gene therapies are revolutionizing treatment for hemophilia A and sickle cell disease; however, they are saddled with hefty price tags and limited patient populations that are impacting uptake.

Research at the annual meeting of the American Society of Hematology evaluated patient and caregiver perspectives on gene therapies for sickle cell disease, which offer great potential but have had slow uptake.

Gene therapies have a lot of potential to improve health outcomes for patients with life-threatening diseases but face a number of barriers that restrict access.

Kebilidi is a one-time gene therapy administered directly to the brain in patients with AADC deficiency, rare genetic disorder.

In a phase 2 trial, a gene therapy reduced angioedema attacks, which could significantly change the quality of life for patients with hereditary angioedema.

In-depth interviews and an online survey found support for gene therapy as a treatment for inherited blood disorders, but also several obstacles that must be addressed.

Up to half of patients showed significant improvement in study results presented at the American Academy of Ophthalmology (AAO) annual meeting

One of two new studies found most patients doing well six years after treatment. Another study found blood cancers in 17% of patients given the therapy using a slightly different protocol.

The Phase I/II trials for rare forms of hereditary blindness further confirmed the potential for gene therapy treatments of congenital diseases of the retina.

“Cure” is not an immutable term, the authors of a new study write, and its meaning depends on the context in which it is used. Sometimes, it shouldn’t be.

The researchers took what they said was a conservative approach when plugging variables such as one-time and future costs into their updated model.

CMS will provide key logistical support to states that participate in its outcomes-based funding model for gene therapy treatment for sickle cell disease.

Gene therapy is enormously complicated, inefficient and slow. Early tests of a newly developed machine learning model show how much faster, and more efficient, it might be.

The key to lowering gene therapy costs – and getting treatments to where they are most needed – could be making them in middle- low-income countries, argue the authors of a recent commentary in Nature.

Humira and Sterlara are still the mainstays of spending, but Vizient that expenditures on drugs that treat genetic disorders will climb to $25 billion in 2026,

The intent is for both parties to share information that will enhance the agency’s efficiency and quality of reviews.

A number of physicians experienced in treating DMD across the nation contributed to this article recently published in the Annals of the Child of Neurology Society.

Denmark has also set up a reimbursement arrangement for Hemgenix, which has a list price of $3.5 million in the U.S.

Gene therapies are incredibly expensive and tend to benefit small numbers of people, a combination that presents myriad financing challenges for patients, providers, payers and producers. A new study explored some of the options.

The application for afami-cell to treat patients with advanced synovial sarcoma is the first engineered T-cell therapy for solid tumors submitted to FDA.

The FDA has approved Lyfgenia and Casgevy to treat patients with sickle cell disease. Casgevy is the first FDA-approved gene therapy to use the CRISPR gene editing technology.

Two investigational therapies for Rett syndrome, using different technologies, aim to control expression of the delivered gene and avoid the adverse events associated with conventional gene therapy.