beta-thalassemia

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

By bringing life-changing benefits to people with rare diseases and cancers, cell and gene therapies are reshaping the field of medicine. These treatments have recently gained tremendous publicity for meeting long-standing unmet needs and being extremely costly. But high production costs are limiting patient access...

Vertex Pharma and CRISPR Therapeutics are the first companies to seek FDA clearance for a gene-editing therapy. Vertex Pharmaceuticals and CRISPR Therapeutics have gotten closer to introducing exagamglogene autotemcel (exa-cel), a one-time treatment for sickle cell disease (SCD) and transfusion-dependent beta-...

The current Beta-thalassemia market is experiencing the emergence of gene therapies as a one-time cure for the patients. Beta thalassemia is relatively a common heterogeneous autosomal recessive hereditary blood disorder. It is a common form of hemoglobinopathy characterized by ineffective erythropoiesis leadi...

Beta thalassemia, globally affects almost 288,000 people, with an incidence of 60,000 cases every year. A rare monogenic genetic disease, beta-thalassemia is caused by more than 200 mutations of the beta-globin gene (HBB). The Beta-thalassemia pharmacological treatment mainly comprises of regular blood-transfus...

Thalassemia is a group of inherited blood disorders that are a result of defects or mutations in the genes. The defect results in defective or less production of the haemoglobin, thus hampering the transport of the oxygen to all the body parts. The most common form of the thalassemia is Beta-Thalassemia. It cau...

The US FDA for the third time has refused to give approval to Lipocine’s oral testosterone drug, Tlando. The drug is an oral testosterone replacement therapy in clinical trials for the treatment of Primary and Secondary hypogonadism in males. The drug contains containing Testosterone Undecanoate (TU) which help...

 Vertex, CRISPR Therapeutics treated a patient using CRISPR In the very first, a patient diagnosed with Beta thalassemia was treated using CRISPR gene editing tool by Vertex and CRISPR Therapeutics.  CTX001 used to treat the disease, was extracted from hematopoietic stem cells from patients, engineered ex...

If dementia genes are found, drug discovery can be accelerated in brain disorders The overproduction of a protein called tau has been detected in the neuron degeneration that causes Alzheimer’s and other forms of dementia. But the exact genetic process that causes tau is still a mystery. Scientists of the University...

Gilead remunerates USD 50 Million to collaborate with synthetic lethality startup Tango Gilead is remunerating USD 50 million upfront to enter into an immuno-oncology collaboration with Massachusetts-based Tango Therapeutics. The deal provides Gilead with the option to receive the worldwide rights to five targets d...