Specific Biologics
Specific Biologics is developing Dualase genome editors, a differentiated platform for in vivo gene therapy that enables precise removal, repair, and insertion of DNA sequences. The editors are small ( 3.7kb), fitting into a single AAV or lipid nanoparticle for delivery, and achieve high specificity with no detectable off-target effects. Pipeline programs target CNS, lung, and liver indications, including repeat removal for neurodegeneration and mutation-agnostic correction for cystic fibrosis.
- Drug Discovery & Therapeutics
- Biotechnology
- Healthcare Technology
Funding
Raised to date
$36.3MRaised 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.
across 4 rounds
ABBSDC+3$35M · Series A
Announced Nov 2026
Disclosed cumulative: $36.3M
Founders
Founder details are not available yet.
Product
Problem
Current in vivo genome editing technologies face significant limitations, including large cargo sizes that hinder efficient delivery, off-target edits that raise safety concerns, and mechanisms that are inefficient for repairing or removing large DNA sequences. These challenges restrict the development of durable, broadly applicable gene therapies for many genetic diseases.
Solution
Specific Biologics is advancing Dualase genome editors, a differentiated platform that uniquely overcomes these challenges through a two-site mechanism. This mechanism enables seamless removal, repair, and insertion of small or large sequences with predictable and efficient edits. The editors are small (~3.7kb), allowing them to be packaged into a single adeno-associated virus (AAV) or delivered as small RNAs in lipid nanoparticles. Dualase leverages a proprietary engineered Cas domain and a two-site requirement to achieve high specificity with no detectable off-target effects. The platform is programmable via targeting RNA and a frequently occurring 'CNNNG' target site, enabling broad in vivo therapeutic applications.
Target Audience
Primary customers are biopharmaceutical companies and research partners seeking to develop in vivo gene therapies for genetic diseases, particularly those targeting CNS, lung, and liver indications.
Features
- Two-site mechanism (I-TevI and Cas domains) that leaves different DNA ends ('sticky' and 'blunt') to bias natural cell repair pathways toward correct outcomes
- Small editor size (~3.7kb) enabling delivery via a single AAV or as small RNAs in lipid nanoparticles and polymers
- Proprietary engineered Cas domain and two-site requirement for high specificity with no detectable off-target edits in vitro or in vivo
- Programmable through targeting RNA and the I-TevI target site 'CNNNG', which occurs frequently in the genome
- Mechanism for precise removal of large (kilobase) sequences via dual-guided I-TevI domains, repaired through the non-homologous end-joining (NHEJ) pathway
- Mechanism for repair or insertion of small (100's of bases) and large (1000 bases) sequences using RNA-templated DNA repair via polymerase theta (Pol θ)
- Pipeline programs in CNS, lung, and liver indications, including repeat removal for neurodegeneration and mutation-agnostic correction for cystic fibrosis