Specific Biologics
Specific Biologics is a Toronto-based preclinical biotech developing the Dualase® dual-cut genome editing platform for in vivo treatment of genetic diseases, with initial pipeline programs in CNS neurodegeneration, cystic fibrosis, and liver indications.
- Company typePrivate
- Founded2017
- HeadquartersToronto, Canada
- Headcount1–10
- GTM typeB2B
- OfferingHardware or Manufacturing
What Specific Biologics does
Specific Biologics Inc. is a Toronto-based, venture-backed preclinical biotechnology company developing the Dualase® platform, a novel class of dual-cutting genome editing enzymes that overcome key limitations of single-cut CRISPR-Cas9 systems. The Dualase architecture combines a programmable I-TevI domain with an engineered Cas domain to produce two site-specific DNA cuts (sticky and blunt ends), which biases cellular repair toward precise outcomes, including scarless insertions, deletions, and base changes via a polymerase theta-mediated RNA-templated pathway. The platform's small (~3.7kb) cargo fits within a single adeno-associated virus (AAV) or can be delivered as small RNAs in lipid nanoparticles (LNPs), enabling broad in vivo therapeutic applicability across CNS, lung, liver, and skeletal muscle tissues.
The company's preclinical pipeline targets repeat expansion disorders (e.g., ALS/C9ORF72, Huntington's Disease) via single AAV delivery to the CNS, cystic fibrosis via a proprietary nebulized LNP formulation for lung delivery, and liver indications via LNP-encapsulated RNA. A machine learning-enabled prediction platform is being co-developed with Western University under a CAD$1.8M Genome Canada/Ontario Genomics grant to accelerate identification of new Dualase editors. Specific Biologics is funded by Lumira Ventures and adMare BioInnovations (seed, December 2021; renewed 2023) and has received non-dilutive awards from the Cystic Fibrosis Foundation Therapeutic Development Award (2021).
The business model is structured for long-horizon value creation through eventual commercialization of Dualase-based therapeutics and/or platform licensing to pharmaceutical partners for co-development in additional indications. Near-term, the company is pre-revenue with 1–10 employees and pursues business development outreach, academic collaborations (Western University), and non-dilutive grant funding to advance pipeline assets toward IND-enabling studies.
Specific Biologics firmographics
Firmographics- Name
- Specific Biologics
- Legal name
- Specific Biologics Inc.
- Website
- https://specificbiologics.com
- Company type
- Private
- Founded year
- 2017
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Specific Biologics is a Toronto-based preclinical biotech developing the Dualase® dual-cut genome editing platform for in vivo treatment of genetic diseases, with initial pipeline programs in CNS neurodegeneration, cystic fibrosis, and liver indications.
- Ownership category
- akta.pro rank
Specific Biologics industry classification
Industry- Product category
- Biopharmaceuticals
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Engineered Protein Therapeutics (de novo/AI-designed proteins, novel scaffolds) (HLAAAAAJ)
- akta.pro secondary industries
- Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization) (HLAAAIAC), Synthetic Biology & Genetic Circuit Design Platforms (pathway engineering, chassis development) (HLAAAIAD), Protein Interaction & Structural Biology Platforms (cryo-EM, X-ray, NMR, biophysics) (HLAAAIAH)
Keywords
Where Specific Biologics is headquartered
LocationHeadquarters
- HQ city
- Toronto
- HQ country
- Canada
- HQ region
- North America
Offices1 record
Markets served
Specific Biologics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales
Revenue model
- Therapeutic Product Sales: Pre-revenue biotech company developing gene editing therapies. Revenue model would be through commercialization of Dualase-based therapeutics for genetic diseases once approved by regulatory authorities.
- Platform Licensing: Potential licensing of Dualase platform technology to pharmaceutical partners for development of gene editing therapeutics in additional indications.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels4 records
Specific Biologics product offering
Product offeringCore offering
Specific Biologics is a preclinical biotechnology company developing the Dualase® genome editing platform, a novel class of enzymes that make two targeted DNA cuts using an I-TevI domain and an engineered Cas domain. The company is advancing a pipeline of in vivo gene editing therapeutics focused on precise removal of pathogenic DNA repeats in CNS, cystic fibrosis (lung), and liver indications, with delivery via single AAV or lipid nanoparticle encapsulated RNAs.
Product overview
Specific Biologics is developing a single unified platform called Dualase® for gene editing therapeutics. The Dualase platform offers two distinct mechanisms of action: (1) precise sequence removal using dual-guided I-TevI domains for large repetitive sequences delivered via single AAV or small RNAs, and (2) repair or insertion of small or large sequences using RNA-templated DNA repair through polymerase theta. The company's pipeline includes programs targeting neurodegeneration (CNS), cystic fibrosis (lung), and liver diseases, with additional discovery-stage candidates in skeletal muscle and other indications.
Differentiator
Problem solved
Functional benefit
Products and services
- Dualase®
Quantifiable outcome
- No detectable off-target effects in vitro or in vivo using targeted and genome-wide surveys
- +2 more outcomes
Companies that use Specific Biologics
Customer profileSegments4 records
Ideal customer profiles2 records
Specific Biologics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
AI capability2 records
Feature7 records
Specific Biologics partnerships and signals
Strategic signalPartnerships
One partnership is on record.
- Western UniversitycoreResearch partnership with Dr. David Edgell's team at Western University to develop machine learning-enabled prediction platform for Dualase genome editors, integrating large-scale genomic datasets and structural modeling. Research team will build computational platform to identify potent Dualase editors for new therapeutic targets.
Scale indicators3 records
Recent moves6 records
Expansion highlights5 records
Specific Biologics competitors and assessment
Company assessmentDirect peers
- Intellia Therapeutics: Clinical-stage in vivo CRISPR-Cas9 gene editing company with programs in transthyretin amyloidosis, hereditary angioedema, and hemophilia. Competes for the same in vivo gene editing opportunity and pharma partnering attention.
- Prime Medicine: Clinical-stage developer of prime editing, a precise gene editing modality that — like Dualase — aims to make targeted insertions, deletions, and base changes without double-strand breaks. Closest mechanistic analog given scarless repair approach.
- Editas Medicine: Clinical-stage CRISPR-Cas9 and Cas12a gene editing company with both in vivo and ex vivo programs. Direct competitor in CNS (e.g., LCA10) and other genetic disease indications.
- Beam Therapeutics: Clinical-stage biotech developing in vivo base editing therapeutics for genetic diseases. Most direct competitor to Specific Biologics given overlapping approach to precise in vivo gene correction, with multiple programs already in the clinic.
- CRISPR Therapeutics: Pioneer CRISPR-Cas9 therapeutic developer with approved Casgevy (sickle cell, beta-thalassemia) and active in vivo programs. Sets the competitive benchmark for the gene editing space and likely acquirer/partner.
- Verve Therapeutics: Clinical-stage in vivo base editing company focused on cardiovascular disease, with lead programs in familial hypercholesterolemia. Competes with Specific Biologics' liver-directed base-change correction approach.
Emerging players
- Shape Therapeutics: Preclinical/early-clinical RNA editing and gene therapy company developing programmable editors for repeat expansion disorders and other genetic diseases. Overlap with Specific Biologics' CNS repeat removal program.
- Korro Bio: Clinical-stage RNA editing company targeting repeat expansion disorders including Huntington's Disease and ALS. Direct overlap with Specific Biologics' CNS neurodegeneration program and likely competitor for partnership capital.
- Tessera Therapeutics: Gene writing company developing technologies to make precise insertions, deletions, and replacements in the genome. Competes conceptually with Dualase's RNA-templated DNA repair approach for scarless sequence insertion.
Broad incumbents
- Vertex Pharmaceuticals: Large biopharma with established cystic fibrosis franchise (Trikafta/Kaftrio) and a partnered CRISPR Therapeutics in vivo program (CTX001 → Casgevy). Most relevant incumbent because of CF indication overlap and proven willingness to partner on gene editing.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat4 records
Key risks5 records
Key highlights6 records
Customer concentration
Specific Biologics social profiles
Digital presenceSpecific Biologics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Specific Biologics leadership team
Management profileNumber of profiles
Profiles8 records
Specific Biologics funding detail
Funding detailFunding overview
Funding rounds4 records
Investors5 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Specific Biologics M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Specific Biologics
What does Specific Biologics do?
Specific Biologics is a preclinical biotechnology company developing the Dualase® genome editing platform, a novel class of enzymes that make two targeted DNA cuts using an I-TevI domain and an engineered Cas domain. The company is advancing a pipeline of in vivo gene editing therapeutics focused on precise removal of pathogenic DNA repeats in CNS, cystic fibrosis (lung), and liver indications, with delivery via single AAV or lipid nanoparticle encapsulated RNAs.
Is Specific Biologics a public or private company?
Specific Biologics is a private company. It is classified as venture growth investor backed and is currently operating.
When was Specific Biologics founded?
Specific Biologics was founded in 2017. It employs 1 to 10 people.
Where is Specific Biologics based?
Specific Biologics is headquartered in Toronto, Canada, in the North America region.
How does Specific Biologics make money?
Two revenue lines are on record. Therapeutic Product Sales are the primary driver. The others are platform Licensing.
Who are Specific Biologics's main competitors?
Direct peers on record are Intellia Therapeutics, Prime Medicine, Editas Medicine, Beam Therapeutics, CRISPR Therapeutics and Verve Therapeutics. Emerging players are Shape Therapeutics, Korro Bio and Tessera Therapeutics. Vertex Pharmaceuticals is listed as a broad incumbent.
Does Specific Biologics have an API?
No public API is recorded for Specific Biologics.
What industry is Specific Biologics in?
Specific Biologics's product category is Biopharmaceuticals. Its primary akta.pro industry code is HLAAAAAJ, Engineered Protein Therapeutics (de novo/AI-designed proteins, novel scaffolds), with a secondary code of HLAAAIAC, Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization). Its NAICS code is 541714 and its SIC code is 2836.