TransformingInsight into Impact

Envisagenics has a validated pipeline of novel drug targets and therapeutics discovered by SpliceCore®.

Oncology

We have developed and de-risked promising clinically actionable assets across multiple indications.

Neurodegenerative diseases (e.g., ALS)

We are advancing splice-switching oligonucleotide therapies toward the clinic.

By focusing on high-impact, disease-specific targets, we aim to deliver safer, more effective therapies to patients with urgent unmet needs.

Target Identification

Splice Core

SpliceCore® is Envisagenics’ proprietary AI platform that discoversnovel, prevalent, disease-specific splicing events with high therapeutic potential. SpliceCore identifies key splicing alterations that drive disease or distinguish diseased cells from healthy ones. SpliceCore cuts through the complexity of splicing to enable new classes of therapeutic targets that traditional methods miss. Our targets have demonstrated strong experimental validation in cancer and neurodegenerative diseases.

SpliceCore Benchmarks

Cutting through transcriptomic complexity to find novel, prevalent, disease-specific targets.

400xlarger search space > 14 million splicing events
10%-82%target prevalence in patients
100%novel disease-specific targets
0%near-zero expression in normal tissues
70%experimental validation rate

Platform Enablers

Driven by a team of expert scientists and engineers, and strengthened by collaborations with leaders in AI and high-performance computing, we have built the robust infrastructure behind SpliceCore to analyze vast, complex datasets with exceptional speed, precision, and scalability.

Cold Spring Harbor LaboratoryNvidia Inception ProgramNational Institute of HealthMemorial Sloan Kettering Cancer CenterMicrosoftThe Jackson Laboratory

Alternative Splicing and Disease

More than 95% of human genes undergo alternative splicing, a process that enables a single gene to produce many different proteins. This mechanism drives the complexity needed for different cell types, tissues, and biological functions. When the splicing process breaks down, it can contribute to serious diseases like cancer and neurodegenerative disorders. Despite its fundamental role in biology, RNA splicing remains largely overlooked in drug discovery.

At Envisagenics, we’re changing that. By focusing on disease-specific splicing events that are also common across patients, we’re uncovering an entirely new class of drug targets, enabling safer, more precise treatments for conditions with urgent unmet needs.

SpliceIO

Envisagenics’ machine learning module for the identification of tumor-specific RNA splicing-derived neoepitopes for the development of first-in-class therapeutic candidates.

SPINRAZA®

Led by Dr. Adrian Krainer, Envisagenics’ founding scientific team helped in the development of SPINRAZA®. In December 2016, SPINRAZA became the first FDA approved RNA therapeutic to treat Spinal Muscular Atrophy (SMA) by correcting the disease-causing splicing error.

Since then, with further advances in RNA therapeutic delivery systems, an increasing number of RNA therapeutics have received or are close to obtaining FDA approval.

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