Expected rapid advances in CAR T and other cellular therapies may expand treatment options not only in cancer but also in ...

The Gene Therapy R&D market presents robust opportunities, driven by clinical advancements in CAR-T therapies and a strong pipeline, particularly for cancer and rare diseases. Despite challenges from ...

An HIV-derived nucleoside therapy now treats rare genetic diseases by restoring mitochondrial DNA and improving muscle ...

CGTxchange leverages the combined capabilities of the organizations in an unprecedented way to engage a broad array of potential partners and funders to efficiently identify, evaluate, finance, ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is an advanced technology developed in 2012 that can be used to edit genes. It can be used to find specific DNA sequences inside ...

Live Science's health channel editor makes predictions about the medical breakthroughs and public health shifts to come in ...

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

Collaboration Aims to Establish Sustainable Access to a Newly Approved Therapy Targeting a Rare Immunodeficiency, Filling Urgent Gap Left by For-Profit Efforts CAMBRIDGE, Mass. and ROME, Dec. 15, 2025 ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...