Rgenta Therapeutics develops oral, small-molecule RNA-targeting medicines aimed at treating previously incurable oncology and neurological disorders. By focusing on RNA regulation, the company addresses the challenge of drugging targets that have been difficult to reach with traditional therapies.
Funding
$8M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many disease-causing proteins are considered "undruggable" by traditional methods, hindering the development of effective treatments for oncology and neurological disorders. Current therapeutic approaches often fail to address the underlying genetic dysfunctions that drive these diseases.
Solution
Rgenta Therapeutics is developing oral, small-molecule RNA-targeting medicines to treat previously incurable diseases by focusing on RNA regulation. Their approach involves analyzing vast genomic data to pinpoint interactions of the splicing machinery and RNA, enabling the design of potent drugs that modulate therapeutic targets. These small molecule "glues" stabilize the interaction between the target RNA and splicing machinery, driving alternative splicing and leading to the degradation of the target RNA and elimination of the pathological target protein. This innovative strategy unlocks new drug possibilities for previously "undruggable" targets, with initial focus on oncology and neurological disorders.
Target Audience
The primary target audience includes patients with oncology and neurological disorders that are currently difficult to treat, as well as pharmaceutical companies seeking innovative RNA-targeting technologies for gene and cell therapies.
Features
- Orally available small molecules designed to target and modulate RNA
- Focus on previously "undruggable" targets in oncology and neurological disorders
- Proprietary platform for identifying and validating RNA targets
- Small molecule "glues" that stabilize the interaction between target RNA and splicing machinery
- RSwitch technology for regulatable control of gene and cell therapies
- Oncology program targeting oncogenic proteins like MYB
- CNS program addressing repeat expansion diseases (REDs) by targeting the root cause of genetic disorders