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Innovation in immuno-oncology: Leading companies in in-vitro T-cell activation

Pharmaceutical Technology

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: In-vitro T-cell activation. Immatics is the leading patent filer in in-vitro T-cell activation.

In-Vitro 130
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Vertex gains FDA approval for expanded use of CF drug Trikafta

Pharmaceutical Technology

The treatment is now indicated for CF patients with a minimum of one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or with a mutation in the CFTR gene that responds to Trikafta based on in vitro data. It was previously approved to treat CF in children aged between six and 11 years.

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Alnylam identifies gene to lower abdominal fat and decrease cardiovascular risk

Pharmaceutical Technology

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

Gene 130
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Vertex secures FDA clearance for infants’ cystic fibrosis therapy

Pharmaceutical Technology

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results. This cohort showed a safety profile identical to that found in older children and adults.

In-Vitro 246
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Ginkgo Bioworks partners with WARF for new GD2 CAR T-cell therapies

Pharmaceutical Technology

All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models. Topic sponsors are not involved in the creation of editorial content.

In-Vivo 264
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Researchers reverse the in vitro and in vivo effects of the mutation that causes Stormorken syndrome

Medical Xpress

They study gene variation and mutations that cause rare genetic diseases. That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. A mutation is a permanent change in the genetic material.

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First hybrid gene therapy shows early promise in treating long QT syndrome

Scienmag

— In a new study published in Circulation, Mayo Clinic researchers provide the first preclinical, proof-of-concept study for hybrid gene therapy in long QT syndrome, a potentially lethal heart rhythm condition. ROCHESTER, Minn.