Gennao Bio develops targeted nucleic acid therapeutics using its proprietary GMAB platform, which employs a non-viral, cell-penetrating antibody to deliver various nucleic acid payloads directly to muscle cells and tumor cells via the ENT2 transporter. This technology addresses the challenge of inefficient delivery mechanisms in genetic medicine, enhancing bioavailability and minimizing off-target effects in the treatment of muscle diseases and cancer.
Funding
$1M 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 genetic medicine approaches face challenges in efficiently delivering nucleic acid payloads to target cells, leading to limited bioavailability and potential off-target effects. This inefficiency hinders the effective treatment of diseases, particularly in muscle and tumor tissues, where targeted delivery is crucial.
Solution
Gennao Bio is developing targeted nucleic acid therapeutics using its proprietary GMAB (Gene Monoclonal Antibody) platform to address the limitations of existing delivery methods. The GMAB platform employs a non-viral, cell-penetrating antibody that specifically targets the ENT2 transporter, which is highly expressed on tumor and skeletal muscle cells. This targeted approach enables direct delivery of various nucleic acid payloads, including single-stranded/double-stranded RNA, mRNA, synthetic RNA, DNA, RNAi and ASOs, into the cell, bypassing the endocytic pathway. By avoiding the endocytic pathway, GMAB enhances bioavailability of the payload in the cell and minimizes off-target effects, improving the therapeutic potential for oncology and rare monogenic skeletal muscle diseases. The GMAB complexes have demonstrated the ability to be delivered into both the cytoplasm and the nucleus.
Target Audience
The primary target audience includes patients with cancer and rare monogenic skeletal muscle diseases, as well as researchers and clinicians seeking more effective and targeted genetic medicine delivery systems.
Features
- Adaptive platform capable of delivering a broad range of nucleic acid payloads (single-stranded/double-stranded RNA, mRNA, synthetic RNA, DNA, RNAi and ASOs).
- Targeted delivery to cells expressing the ENT2 transporter, such as tumor cells and skeletal muscle.
- Non-endosomal uptake mechanism to increase bioavailability of the payload.
- Ability to deliver nucleic acid payloads into both the cytoplasm and the nucleus.
- Potential for repeat dosing with the ability to escalate and titrate dose.
- Scalable and reproducible manufacturing process based on an established antibody pathway.