Beam Therapeutics Announces Clearance of Clinical Trial Authorisation Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) - Seite 2

About Alpha-1 Antitrypsin Deficiency (AATD)
AATD is an inherited genetic disorder that can cause early onset emphysema, liver disease and panniculitis. A severe form of AATD arises when a patient has the p.Glu366Lys (also referred to as E342K) point mutation in both copies of the SERPINA1 gene. This results in the expression of the pathogenic PiZ variant of alpha-1 antitrypsin (Z-AAT) that misfolds and aggregates inside liver cells causing liver damage, rather than being secreted. The Z-AAT that is made in the liver is approximately two-fold less effective in inhibiting neutrophil elastase and poorly secreted into circulation, leading to circulating levels of AAT that are 10-15% of normal in homozygous individuals. As a consequence, the lung is left unprotected from neutrophil elastase and other damaging proteases that can cause progressive, destructive changes in the lung, such as emphysema, and lead to severe lung disease and the need for lung transplantation. The accumulation of Z-AAT protein in the liver can cause liver inflammation, hepatocyte injury and cirrhosis, which can result in liver failure or cancer and, in some patients, require liver transplantation. In addition, circulating Z polymers have increasingly been recognized as a factor contributing to disease severity in the lungs.

It is estimated that approximately 100,000 individuals in the United States (U.S.) have two copies of the Z allele, known as the PiZZ genotype, although only 10-15% of all patients are thought to have been diagnosed. There are currently no curative treatments approved for patients with AATD, and the only approved therapy in the U.S., intravenous AAT protein replacement, has not been shown to prevent ongoing lung function decline and destruction in patients.

About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.