SpliSense develops antisense oligonucleotide (ASO) therapies delivered via inhalation to target genetic diseases caused by splicing mutations and various pulmonary conditions. Their approach aims to restore protein functionality in patients with unmet medical needs, providing a non-invasive treatment option for previously untreatable illnesses.
Funding
$43M 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.
CFFounders
Product
Problem
Many genetic diseases and pulmonary conditions are caused by splicing mutations that disrupt normal protein production. Current treatment options are limited, particularly for patients with rare or previously untreatable illnesses affecting the lungs. Invasive procedures are often required to deliver therapies directly to the affected tissues.
Solution
SpliSense is developing antisense oligonucleotide (ASO) therapies designed to correct splicing mutations and restore protein functionality in patients with genetic and pulmonary diseases. Their approach utilizes RNA-based treatments delivered non-invasively via inhalation directly to the lungs. By targeting the root cause of these diseases at the RNA level, SpliSense aims to provide transformative therapies for conditions with significant unmet medical needs. The company's pipeline includes a variety of therapies in different stages of development, from discovery to IND.
Target Audience
The primary target audience includes patients with genetic diseases caused by splicing mutations and individuals suffering from various pulmonary conditions with limited treatment options.
Features
- Antisense oligonucleotide (ASO) technology to target specific RNA sequences
- Non-invasive delivery via inhalation for direct targeting of lung tissue
- RNA-based treatments designed to correct splicing mutations
- Pipeline of therapies in various stages of development, from discovery to IND