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The European Union's health regulator on Friday said it had recommended granting a conditional marketing authorisation for a gene therapy by Australian drugmaker CSL Ltd and partner uniQure N.V. , to treat haemophilia B, a rare bleeding disorder which is caused by genetic anomalies. About one in 40,000 people are affected by the inherited disorder, caused by a gene mutation that hampers the body's ability to make clotting protein factor IX. If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said. CSL had acquired exclusive global rights to Hemgenix in May last year from uniQure N.V.

Capitalising on strong demand for its obesity therapies, Novo Nordisk's growing appetite for deals has fuelled a bet on a U.S. gene-editing company called Life Edit Therapeutics. The Danish drugmaker's collaboration with Durham, North Carolina-based Life Edit Therapeutics - owned by ElevateBio, a cell and gene therapy company in Waltham - is focused on up to seven programs for rare genetic disorders as well as cardiometabolic diseases. At the heart of the tie-up is Life Edit's technology, called base editing, which is designed to make precise changes to the human genome by tweaking one base - or letter - into a different one without affecting other letters. Single-letter mistakes, called point mutations, can give rise to genetic diseases.

'Tepotinib' has been recommended by the National Institute for Health and Care Excellence (NICE) to patients with a specific gene mutation of advanced non-small-cell lung cancer (NSCLC). The new potentially life-extending drug is available from Thursday (14 April). It has been recommended for routine use across the NHS in England through Project Orbis, a programme to review and approve promising cancer drugs helping patients access treatments faster. NICE has published its final appraisal document recommending tepotinib (also known as Tepmetko and produced by company Merck Serono Ltd) as an option for treating advanced NSCLC with METex14 skipping gene alterations in adults. People with METex14 skipping alterations of NSCLC make up between 1-2% of all adults with lung cancer in England. Tepotinib, which requires people to take two tablets once daily, provides a new targeted treatment for adults with METex14 skipping gene alterations. Just over 700 people in England would be eligible to receive tepotinib as either a first or second-line treatment.

The Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised an innovative and first-of-its-kind gene-editing treatment for sickle-cell disease and transfusion-dependent β-thalassemia. Known as Casgevy (exagamglogene autotemcel), the treatment is based on the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. Casgevy is the first medicine to be licensed that uses CRISPR, and it is to be used for treating patients aged 12 and over. To date, a bone marrow transplant has been the only permanent treatment option for these life-long conditions, which in some cases can be fatal. The MHRA's authorisation came after a rigorous assessment of its safety, quality and effectiveness.

The new deal struck by the NHS will enable provision of a life-saving treatment for babies and young children who suffer with a rare and fatal genetic disease, metachromatic leukodystrophy (MLD). The revolutionary gene therapy treatment, known by its brand name Libmeldy, is used to treat MLD, which causes severe damage to the child's nervous system and organs, leading to a life expectancy of just five to eight years. Having a reported list price of more than £2.8 million, it is the most expensive drug in the world, but can now be offered to young patients on the NHS in England after the health service negotiated a significant confidential discount. the drug works by removing the patient's stem cells and replacing the faulty gene that causes MLD before then re-injecting the treated cells into the patient. The most common form of MLD usually develops in babies younger than 30 months and can cause loss of sight, speech and hearing, as well as difficulty moving, brain impairment, seizures, and eventually death.

A recent study of researchers in the UK have discovered a gene responsible for regulating the consumption of alcohol. This study is based on experiment, centered on the drinking habits of mice.

A recent study of researchers in the UK have discovered a gene responsible for regulating the consumption of alcohol. This study is based on experiment, centered on the drinking habits of mice.

There is no sign of a surge of the Omicron coronavirus variant in Britain so far, the chief of the UK Health Security Agency said on Tuesday, adding that there was no rise in the proportion of tests with a quirk that distinguishes it from Delta. Jenny Harries said there were five confirmed Omicron cases in England and 10 "highly likely" cases, but that was not a sign of an increase in "S-gene target failure" in PCR tests in Britain, which is a feature of Omicron. "Right across the country we watch for… S-gene target failure, which is a sort of proxy measure," Harries told BBC radio. Cases with S-gene target failure can then be prioritised for full genomic sequencing.

RNA interference (RNAi) is the process by which the expression of a target gene can be effectively silenced or knocked down.

The global gene delivery technologies market size is expected to reach USD 7.9 billion by 2028

The global gene therapy market size is expected to reach USD 10.0 billion by 2028 registering a CAGR of 20.4% during the forecast period

Module 1 - Understanding the clinical value of genes and molecules identified in the mechanism of disease is organized by Persona Biomed, Inc

Majority of the most common forms of kidney cancer (clear cell renal cell carcinoma, ccRCC) involves a mutation of the same gene, called PBRM1

A healthy diet with lots of fruits and vegetables can significantly weaken the effect of a gene associated with an increased risk of heart disease, a new study suggests.

A revolutionary discovery by the researchers have proved that the most severe form of malaria, caused by the parasite Plasmodium falciparum,is linked with natural variation in human red blood cell genes.

A revolutionary discovery by the researchers have proved that the most severe form of malaria, caused by the parasite Plasmodium falciparum,is linked with natural variation in human red blood cell genes.

Supports a normal, healthy Inflammatory Response. Reactivates the joint health gene IGF-1 in Human Chondrocytes, Supports normal cartilage synthesis.

The University of Queensland researchers have contributed to the discovery of three new genes, thus opening the opportunity for targeted treatments of motor neuron disease (MND) as these genes increase the risk of MND. The study is published in Nature Genetics.