SpliSense Overview
SpliSense is a clinical stage company developing transformative RNA-based treatments for unmet needs relating to diverse pulmonary diseases. Its treatment is based on clinically validated Antisense Oligonucleotide (ASO) technology, delivered non-invasively by inhalation to the lungs. The company is focused on a variety of innovative therapies, in different development stages from discovery to IND. Using an innovative approach, SpliSense aims to target the root cause of the disease, to overcome specific genetic mutations and modulate proteins expression and function. The company recieved FDA approval for its lead ASO product, SPL84, for the treatment of patients with cystic fibrosis.
Cumulative Funding Raised Over Time ($)
Latest News
growth-positive
/PRNewswire/ -- SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced...
SpliSense, a clinical-stage biotechnology company, announced positive results from its Phase 2 study of SPL84, an antisense oligonucleotide therapy for cystic fibrosis (CF). The study showed improvements in lung function in up to 70% of participants, with no safety concerns identified. This marks the first evidence of potential clinical benefit of antisense oligonucleotide therapy in a pulmonary disease. The results validate SpliSenses platform, supporting the advancement of additional pulmonary programs into the clinic by early 2026. SPL84 has been granted Orphan Drug and Fast Track designations by the FDA, highlighting its potential to address unmet medical needs in CF treatment.
Product StageFDA approved/pending approval
growth-positive
SpliSense Receives FDA Fast Track Designation for SPL84 for the Treatment of Cystic Fibrosis
SpliSense, a biotechnology company specializing in RNA-based therapies for pulmonary diseases, announced that its lead product, SPL84, has received Fast Track Designation from the U.S. Food and Drug Administration (FDA). SPL84 is designed to treat cystic fibrosis (CF) patients with a specific genetic mutation. This designation will expedite the development and review process, potentially speeding up the delivery of this treatment to patients. The Fast Track status acknowledges the urgent need for effective treatments for CF, particularly for those with the 3849+10 Kb C->T mutation. SpliSenses approach involves using antisense oligonucleotides (ASOs) to correct mutations in the CFTR gene, aiming to restore normal protein function. SPL84 is currently in a global Phase 2 study.
Growth-Positive
SpliSense Announces FDA and EMA Grant Orphan Drug Designation to SPL84-23-1 for the Treatment of Cystic Fibrosis
Biopharmaceutical company SpliSense has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for SPL84-23-1, a therapy for cystic fibrosis (CF) and other pulmonary diseases. The ODD status, granted to drugs intended to treat rare diseases, provides companies with development incentives including tax credits for clinical trials, exemptions from FDA application fees, and potential marketing exclusivity for seven years following regulatory approval. SpliSense plans to commence clinical trials for SPL84-23-1 in mid-2022 in the US, Europe, and Israel.
CustomersPartners
growth-positive
Israeli firm SpliSense moves to clinical trials for cystic fibrosis treatment
SpliSense, an Israeli biopharmaceutical company, announced that its cystic fibrosis treatment technology has received orphan drug designation from both the US FDA and the European Medicines Agency. This designation is a significant milestone for the company, as it aims to address the unmet needs of cystic fibrosis patients. The treatment, SPL84-23, is an antisense oligonucleotide designed to correct a specific mutation in the CFTR gene. The Cystic Fibrosis Foundation invested $8.4 million in SpliSenses technology, part of a $28.5 million funding round. The company plans to initiate clinical studies in the US, Europe, and Israel later this year.
Investment
growth-positive
Inhaled drug could treat rare cystic fibrosis mutations
Jerusalem-based biotech company SpliSense is developing a new treatment for cystic fibrosis using mRNA technology. The companys technology manipulates defective messenger RNA to generate a fully functioning protein, offering hope for the 20% of CF patients with different genetic mutations. SpliSense has raised $28.5 million in a Series B round to fund clinical trials and the development of treatments for other pulmonary ailments. The treatment, based on Anti Sense Oligonucleotide (ASO), is meant to be inhaled and administered weekly for 10 minutes. SpliSense aims to address orphan conditions and potentially benefit the other 80% of CF patients. The first human clinical trials are planned for 2022.
Customers
| Sector | Health Tech & Life Sciences |
Funding
| Total funding | $41.5M |
| Last funding | $12M |
| Stage | B |
| Rounds | 4 |
| Investors | 6 |
Team Members
3
Employees: 11-50
Web & Social Links
| Website | splisense.com/ |
| Careers | Careers page |
Locations
Kalman Ya'akov Man St, Jerusalem, Israel
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