Splisense

What Splisense does

Splisense is a clinical-stage biotechnology company founded in 2017 and headquartered in Jerusalem, Israel, that develops inhaled antisense oligonucleotide (ASO) therapies targeting the genetic root causes of pulmonary diseases. Its proprietary platform deploys short, precisely targeted synthetic RNA strands that bind to specific sequences within target RNA molecules to modulate splicing, correct mutations, or lower gene expression, delivered via inhalation for direct pulmonary penetration with minimal systemic exposure. The company is led by CEO Dr. Gili Hart and scientific founder Prof. Batsheva Kerem of Hebrew University, who was part of the team that discovered and cloned the CFTR gene.

The pipeline comprises four core programs. SPL84, the lead asset, is an inhaled ASO for cystic fibrosis patients carrying the 3849+10kb C->T splicing mutation; it prevents inclusion of 84 intronic nucleotides as a cryptic exon to generate fully functional CFTR protein, and has completed Phase 1 with positive Phase 2a results (10 percentage point ppFEV1 improvement versus placebo, 70% responder rate) and is now in Phase 2b across sites in the US, Europe, and Israel. SPL5AC lowers MUC5AC expression to address muco-obstructive diseases including COPD, asthma, and non-CF bronchiectasis. SPL5B lowers MUC5B expression in idiopathic pulmonary fibrosis to attenuate disease progression. SPL23 targets the W1282X CF mutation via exon-skipping and is in IND-enabling stages. SPL84 has received FDA and EMA Orphan Drug Designation plus EMA e-PRIME designation.

The business model is pre-revenue; the company funds operations through venture capital and non-dilutive grants, primarily from the Cystic Fibrosis Foundation, which has committed approximately $29.9 million across the Series B and two subsequent funding agreements. Future revenue is expected to come from product sales following regulatory approval of approved indications, with eventual distribution likely through specialty pharmacy channels for rare disease therapeutics. Splisense's go-to-market motion is enterprise-field sales oriented, targeting rare CF patient populations through multi-region clinical trials and engaging regulators (FDA, EMA) and large-pharma partners for potential commercialization.

Name

Splisense

Legal name

SpliSense LTD.

Website

https://splisense.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

11–50 employees

Short description

Splisense is a clinical-stage Israeli biotechnology company developing inhaled antisense oligonucleotide (ASO) therapies for genetic pulmonary diseases, primarily targeting cystic fibrosis patients with splicing mutations unresponsive to existing CFTR modulator treatments.

Ownership category

akta.pro rank

Product category

Antisense Oligonucleotide Therapies

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Pulmonary Hypertension & Rare Pulmonary Disease Therapies (HLAIAIAG)

akta.pro secondary industry

COPD & Chronic Bronchitis Management (HLAKANAE)

Keywords

• Antisense oligonucleotide therapy
• RNA-based therapeutics
• Cystic fibrosis treatment
• Pulmonary disease therapeutics
• Splicing mutation therapies

Headquarters

HQ city

Jerusalem

HQ country

Israel

HQ region

Middle East

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Others

Revenue model

1. RNA-based therapeutic development and commercialization: Pre-revenue clinical-stage biotechnology company developing RNA-based ASO therapies for pulmonary diseases. Revenue will be generated through product sales following regulatory approval. The company is funded through venture capital investments and non-dilutive funding from the Cystic Fibrosis Foundation.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

SpliSense is a clinical-stage biotechnology company developing inhaled antisense oligonucleotide (ASO) therapies that target genetic diseases caused by splicing mutations, primarily in pulmonary indications. Its lead candidate SPL84 is an inhaled ASO that addresses the 3849+10kb C->T splicing mutation in cystic fibrosis by preventing inclusion of 84 intronic nucleotides as a cryptic exon, restoring functional CFTR protein. Additional pipeline programs (SPL5AC, SPL5B, SPL23) target muco-obstructive pulmonary diseases, idiopathic pulmonary fibrosis, and the W1282X CF mutation, respectively.

Product overview

SpliSense is a clinical-stage biopharmaceutical company developing a portfolio of inhaled antisense oligonucleotide (ASO) therapies for pulmonary diseases. The core platform utilizes short, precisely targeted proprietary RNA stretches called ASOs to modulate specific mRNA sequences, either correcting genetic mutations or reducing protein expression. The lead product SPL84 targets the 3849+10kb C->T splicing mutation in cystic fibrosis, while SPL5AC addresses muco-obstructive diseases by lowering MUC5AC expression, and SPL5B targets idiopathic pulmonary fibrosis by reducing MUC5B. SPL23 is in development for the W1282X CF mutation. All therapies are delivered via inhalation for direct lung delivery with minimal systemic exposure.

Differentiator

Problem solved

Functional benefit

Products and services

• SPL84 Inhaled antisense oligonucleotide (ASO) therapy for cystic fibrosis patients carrying the 3849+10kb C->T splicing mutation in the CFTR gene. Prevents inclusion of 84 intronic nucleotides as a cryptic exon, generating fully functional CFTR protein. Phase 1 completed, Phase 2 ongoing with positive Phase 2a results showing up to 10 percentage point ppFEV1 improvement versus placebo.
• SPL5AC Inhaled ASO drug targeting MUC5AC mucin expression for treatment of muco-obstructive diseases including COPD, asthma, non-CF bronchiectasis, and primary ciliary dyskinesia. Lowers MUC5AC expression and production, reduces mucus viscosity, eliminates accumulation in airways, and reduces infection and inflammation. Validated in three proof-of-concept animal models.
• SPL5B Inhaled ASO drug for idiopathic pulmonary fibrosis (IPF) that lowers MUC5B expression and production to avoid accumulation of MUC5B mucin and plugs in distal lung regions. Aims to slow or delay IPF progression by addressing mucus-driven fibrotic mechanisms in the distal lung.
• SPL23 Inhaled ASO treatment for W1282X CF mutation that induces skipping over exon 23 in the CFTR transcript, bypassing nonsense-mediated RNA decay (NMD) to produce mature and active CFTR protein. Currently in IND-enabling stages.

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

One partnership is on record.

• Yissum (Hebrew University Technology Transfer)coreTechnology or IntegrationTechnology licensing agreement for ASO technology developed by Prof. Batsheva Kerem from Hebrew University of Jerusalem. Yissum is the technology transfer office of Hebrew University that licensed the foundational intellectual property for SpliSense's platform.

Scale indicators8 records

Recent moves6 records

Expansion highlights6 records

Market position

Competitive moat4 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/splisenseltd

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds4 records

Investors5 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Splisense