Apertura Gene Therapy is developing AAV gene therapies through advanced capsid and payload engineering to enhance gene delivery and expression for patients with rare diseases. The company addresses the limited treatment options available for debilitating conditions by creating targeted genetic medicines that improve therapeutic outcomes.
Funding
$67M 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 rare diseases lack effective treatments due to the challenges of delivering gene therapies to the appropriate tissues and achieving sufficient gene expression. Current gene therapy approaches often face limitations in targeting specific cells, crossing biological barriers, and ensuring long-lasting therapeutic effects.
Solution
Apertura Gene Therapy is developing next-generation AAV (adeno-associated virus) gene therapies by engineering both the viral capsid and the therapeutic payload. The company's approach aims to overcome limitations in gene delivery and expression, enhancing the potential for treating a range of rare diseases. By designing novel AAV capsids, regulatory elements, and payloads, Apertura seeks to improve the targeting of specific tissues, enhance gene transfer efficiency, and optimize therapeutic outcomes for patients with limited treatment options. Their technology has the potential to increase the accessibility and impact of gene therapy for caregivers and patients.
Target Audience
The primary target audience includes patients with rare genetic diseases, their caregivers, and corporate and academic collaborators focused on advancing gene therapy.
Features
- AAV capsid engineering to improve tissue-specific targeting and transduction efficiency.
- Payload engineering to optimize gene expression and therapeutic protein production.
- Development of CNS-targeted capsids for neurological disorders.
- Development of capsids that target both the CNS and muscle tissue for neuromuscular diseases.
- Machine learning-powered delivery and expression platforms.