Elixirgen Therapeutics develops RNA-based therapies using proprietary technologies such as Bobcat mRNA™ and controllable self-replicating RNA (c-srRNA™) to deliver large genetic payloads and control gene expression, addressing genetic diseases like Duchenne muscular dystrophy and telomere biology disorders. Their approach enables the encoding of full-length dystrophin and the extension of telomeres, providing potential treatments for conditions with significant unmet medical needs.
Funding
$7M 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
Current gene therapy vectors have payload size limitations, restricting their ability to deliver large genes required for treating certain genetic diseases. For example, adeno-associated virus (AAV) vectors have a maximum payload capacity that is insufficient for delivering the full-length dystrophin gene, which is mutated in Duchenne muscular dystrophy (DMD). This limitation necessitates the development of alternative delivery methods capable of carrying larger genetic payloads.
Solution
Elixirgen Therapeutics is developing RNA-based therapies utilizing its proprietary Bobcat mRNA™ and controllable self-replicating RNA (c-srRNA™) technologies to overcome payload size limitations and control gene expression. The Bobcat mRNA™ technology enables the delivery of large genetic payloads, such as the full-length dystrophin gene for DMD treatment, while c-srRNA™ technology allows for controlled and extended gene expression, minimizing off-target effects. Additionally, their ZSCAN4 technology, delivered via the RNA-based Sendai virus, aims to extend telomeres in patients with telomere biology disorders (TBDs). These technologies offer potential treatments for genetic diseases with significant unmet medical needs.
Target Audience
The primary target audience includes patients with genetic diseases such as Duchenne muscular dystrophy and telomere biology disorders, as well as pharmaceutical companies seeking partnerships for RNA-based therapeutic development.
Features
- **Bobcat mRNA™:** Proprietary mRNA design capable of delivering payloads exceeding 13 kb, enabling the encoding of full-length genes like dystrophin.
- **c-srRNA™:** Controllable self-replicating RNA technology that allows for elevated and extended gene expression with an "off switch" to prevent off-target effects.
- **ZSCAN4 Technology:** Utilizes the RNA-based Sendai virus to deliver ZSCAN4, extending telomeres in stem cells through a telomerase-independent mechanism.
- **DMD Program:** Bobcat mRNA™ encoding full-length dystrophin, demonstrating restoration of muscular function in preclinical DMD models.
- **TBD Program:** Phase I/II clinical trial using ZSCAN4 delivered by Sendai virus as an autologous cell therapy, showing evidence of _ex vivo_ telomere elongation in TBD patients.