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CF is a progressive geneticdisease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. These mutations can vary in severity and impact on CFTR function, from complete protein dysfunction to defects in protein folding, trafficking or regulation.
This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction. BridgeBio has been highly active in the geneticdiseases space.
If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the lifesciences. The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing.
Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the LifeSciences. To develop CRISPRoff, the researchers generated a single “dead” Cas9 fusion protein capable of mediating DNA methylation and repressive histone modifications, but devoid of any DNA cleavage function.
DEB patients are also at an increased risk of aggressive skin cancer.DEB usually presents at birth and is caused by one or more mutations in the COL7A1 gene, which encodes type VII collagen (COL7), an important protein that helps connect, strengthen and stabilize the outer and middle layers of the skin.
TSC is a rare geneticdisease that affects approximately 1 in 6,000 people. The disease is caused by mutations on the TSC1 and TSC2 genes, which produce the proteins hamartin and tuberin, respectively.
Hympavzi reduces the activity of TFPI, a naturally occurring anticoagulation protein. Hympavzi is the first anti-tissue factor pathway inhibitor (anti-TFPI) approved in the US for hemophilia A or B, and it’s also the first hemophilia therapy to be administered via a pre-filled auto-injector pen.
December is bringing frost in the North and plenty of cold hard cash for these lifesciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth lifesciences fund. Through its Shielded Living Therapeutics platform, the company is developing functional cures for chronic diseases.
The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. It also affects Hispanic Americans, but at a lower prevalence. The mutation causes red blood cells to develop a crescent or “sickle” shape.
Dietary changes, including restriction of salt and animal protein, are also recommended. Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males.
The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. What’s been shown in mouse models of Rett syndrome which also have the geneticdisease, is that trofinetide helps strengthen those connections between the neurons. Daybue, also called trofinetide, is a tripeptide.
Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the LifeSciences. To develop CRISPRoff, the researchers generated a single “dead” Cas9 fusion protein capable of mediating DNA methylation and repressive histone modifications, but devoid of any DNA cleavage function.
These attacks can be unpredictable, often leading to life-threatening situations when they affect the throat or lungs. HAE is caused by a deficiency or dysfunction of the C1-inhibitor, a protein involved in regulating inflammation. HAE affects approximately one in 50,000 people globally, and there is currently no cure for the disease.
Miplyffa primarily slows disease progression using a different mechanism of action, targeting heat shock protein responses to assist cells under stress.
The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.
Bolstered by world-class R&D capabilities, a highly productive scientific platform and an established management team, Alpine is dedicated to crafting first- or best-in-class multifunctional immunotherapies through distinctive protein engineering technologies, all aimed at enhancing the quality of life for patients.
Alpha-mannosidosis is an extremely rare genetic metabolic disease affecting approximately one in 500,000 people. The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues).
FCS also severely impacts quality of life, causing chronic fatigue and recurrent stomach pain. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. People with FCS often have triglyceride levels higher than 880 mg/dL, compared to a healthy target level of below 150 mg/dL.
Apitegromab is a monoclonal antibody that selectively binds to and inhibits myostatin, a protein that limits muscle growth. Current therapies mainly address motor neuron loss, leaving a need for treatments that directly target muscle weakness.
Moreover, AI has the potential to identify features and signs of disease that may be overlooked by human experts, offering a new layer of diagnostic capability. Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets.
HAE is a rare genetic disorder characterized by recurrent episodes of severe swelling (angioedema) in various parts of the body, including the skin, gastrointestinal tract and airways. Without proper treatment, HAE attacks can be painful, debilitating and potentially life-threatening, especially if they involve the airway.
Mirdametinib is an oral MEK inhibitor that targets the MEK1 and MEK2 proteins, which are part of the RAS/MAPK pathway implicated in the development of NF1-associated tumors. In 2020, AstraZeneca and Merck won FDA approval for Koselugo (selumetinib) to treat NF1-PNs, but its approval was limited to pediatric patients.
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