Adrenas Therapeutics is focused on developing an investigational gene therapy aimed at treating individuals with Classic Congenital Adrenal Hyperplasia (CAH), a genetic disorder that impairs the adrenal glands' ability to produce essential hormones like cortisol and aldosterone. Their gene therapy, known as BBP-631, utilizes adeno-associated virus (AAV) as a delivery vehicle to introduce a functional copy of the CYP21A2 gene, which is crucial for producing the enzyme 21-hydroxylase. This enzyme is necessary for the synthesis of cortisol and aldosterone, and the therapy aims to enable patients to naturally produce these hormones after a single treatment, potentially alleviating the need for lifelong steroid replacement therapies that can have significant side effects.

The approach taken by Adrenas Therapeutics represents a significant advancement in the treatment of CAH, as it seeks to address the underlying genetic cause of the disorder rather than merely managing its symptoms. Preclinical studies have shown promising results, including improved weight gain and reduced hormone levels in animal models, indicating the potential for a durable treatment effect. The company has received Fast Track Designation from the FDA, highlighting the urgency and importance of their clinical trials in providing a new therapeutic option for patients suffering from this condition.

United States

Adrenas Therapeutics is a company that was created with a single mission: to work with scientists, physicians, and patients in developing a gene therapy for people affected by CAH. Adrenas is a subsidiary of BridgeBio, a public company dedicated to finding, developing, and delivering breakthrough medicines for genetic diseases to patients as quickly and safely as possible.

Adrenas Therapeutics

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