Takeda and Arrowhead collaborate to develop and commercialize ARO-AAT
Takeda and Arrowhead Team Up to Unveil Active Liver, Targeted to the Alpha-1 Community.
“This agreement also supports the strategy of selectively partnering to continue investing in the targeted RNAi molecule (TRiMTM) platform”.
They also come together to manage the growing portfolio of RNAi therapeutics targeting various types of tissues.
Both pharmaceutical companies, united for the common good
Takeda’s global presence and expertise in rare disease and GI therapy, along with its long history of serving the Alpha-1 community, make it the ideal partner for ARO-AAT.
Arrowhead thinks Takeda is very well positioned to work with the patient and the medical community.
Remember that there is no treatment to cure patients with alpha 1 antitrypsin deficiency.
What can we say about the AATD?
Alpha-1 antitrypsin associated deficiency (DAAT) is a rare genetic disorder associated with liver and lung disease in children and adults.
It is estimated to affect 1 in 3,000 to 5,000 people in the United States; and 1 in 2,500 in Europe.
The AAT protein is synthesized and secreted mainly by hepatocytes in the liver, its function is to inhibit enzymes that can degrade normal connective tissue.
The most common disease variant, the Z mutant, has a single amino acid substitution that results in improper protein folding.
The mutant protein cannot be secreted efficiently and accumulates in blood cells within hepatocytes.
This triggers ongoing damage to hepatocytes, leading to fibrosis, cirrhosis, and increased risk of hepatocellular carcinoma.
In search of the cure
Individuals with the homozygous PiZZ genotype have a severe deficiency of functional AAT that leads to lung disease and liver disease.
Lung disease is often treated with AAT augmentation therapy, however augmentation therapy does nothing to treat liver disease.
In fact, there is no specific therapy for liver manifestations; therefore, there is a significant unmet need.
Currently, the only available cure is liver transplantation, with its attendant morbidity and mortality.
About ARO-AAT
ARO-AAT está diseñado para reducir la producción hepática de la proteína mutante alfa-1 antitripsina (Z-AAT), he cause of progressive liver disease in patients with AATD.
Reducing the production of the inflammatory protein Z-AAT is expected to halt the progression of liver disease and potentially allow it to regenerate and repair itself.
TAKEDA specialist in gastrointestinal diseases
“We believe that gastrointestinal and liver diseases are not just life-altering conditions, but diseases that can affect a patient’s quality of life.”
With nearly 30 years of experience in gastroenterology, Takeda has made significant advancements addressing the needs of gastrointestinal patients, with treatments for inflammatory bowel disease (IBD), acid-related diseases, short bowel syndrome (SBS) and disorders of motility.
Significant advances are being made that seek to bridge the gap in new areas of unmet need, for patients with celiac disease, eosinophilic esophagitis, alpha-1 antitrypsin associated liver disease, Crohn’s disease, and acute pancreatitis, among others.
ARROWHEAD specialist in genetic diseases
Arrowhead Pharmaceuticals develops drugs that treat intractable diseases by silencing the genes that cause them. These use a wide range of RNA chemicals and efficient modes of administration. Arrowhead therapies activate the RNA interference mechanism to induce rapid, deep, and long-lasting removal of target genes.
RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein.
Arrowhead’s RNAi-based therapies take advantage of this natural gene silencing pathway.
