Gene Editing Tools & Discovery Services
Structured to advance your therapeutic programs.
// Gene Editing &
Engineering R&D Services
Gene Editing Services to Maximize the Clinical Success of Your Therapeutic Program
From demonstrating initial feasibility to engineering clinical candidates and building entire therapeutic pipelines, our platform is designed to accelerate your program and increase its probability of success.
- The Right Tool for Every Target: Our full spectrum of gene editing modalities – including nucleases, base editors, RT editors, large gene insertion and epigenetic editors – ensures we apply the optimal technology to your specific disease target, not a one-size-fits-all solution.
- Centralized Ownership, Streamlined Licensing: Our gene editing modalities and components are wholly owned providing a straightforward path to accessing our technology.
- Proven Track Record: With a demonstrated success rate in generating highly potent and specific gene editors across multiple therapeutic areas, our expertise translates directly into higher quality development candidates for your programs.
- Collaborative Excellence: We are your dedicated partner. You get transparency, collaborative problem-solving, and high-quality data packages designed to support key decisions and regulatory filings.
Learn more about our flexible partnership models:
Rapid Proof of Concept
We typically deliver up to 3 gene editors for your target in under 6 months.
Clinical Candidate Optimization
Leverage our expertise and track record to refine the potency and specificity of your candidate and maximize the likelihood of clinical success.
Build Your Pipeline
This full collaboration model allows you to pursue multiple targets in parallel accessing any or all of our five editing modalities and tapping into our scientific expertise as you advance your R&D efforts.
Maximizing the Probability of Success in Therapeutic Gene Editing Programs
Move programs forward with velocity and confidence.
Working in partnership with your R&D team, ElevateBio helps turn complex biological challenges into viable therapeutic options by providing access to advanced gene editing technology and expertise. Developing genomic medicines requires more than powerful editing tools – it requires a clear way to consistently generate the right data, make informed decisions, and advance programs with confidence.
To support this progression, we offer three structured paths that reflect how research and development teams move programs forward. Each path addresses a distinct stage of decision-making – from establishing feasibility at a single target, to creating promising clinical candidates, to building differentiated pipelines.
Learn more about ElevateBio’s patented gene editing tools and discovery approach in Quickly Finding the Edit That Works: A Smarter, Faster Path to Feasibility in Genomic Medicine.
Three Paths to Therapeutic Progress
| Proof of Concept | Clinical Candidate | Pipeline Model | |
|---|---|---|---|
| Primary purpose | Prove the edit works | Advance candidates toward clinic | Build and scale pipelines |
| Best fit for | Early program evaluation | IND-bound programs | Multi-asset organizations |
| Target scope | Single target | Single target | Multiple targets |
| Problem addressed | Edit feasibility | Refine potency and specificity | Portfolio scale and differentiation |
| Data generated | Feasibility and initial safety | In vitro, In vivo and IND-supporting | Program- and portfolio-level |
| Timeframe | 3-6 months | 18 months or less | Varies based on scope |
| Decision enabled | Go / no-go on edit | Advance toward IND | Pipeline-level commitment |
| Deliverable | 1-3 editors for your target gene | Clinical-ready editors | Clinical-ready editors for multiple targets |
| Proof of Concept |
|---|
| Primary purpose Prove the edit works |
| Best fit for Early program evaluation |
| Target scope Single target |
| Problem addressed Edit feasibility |
| Data generated Feasibility and initial safety |
| Timeframe 3-6 months |
| Decision enabled Go / no-go on edit |
| Deliverable 1-3 editors for your target gene |
| Clinical Candidate |
|---|
| Primary purpose Advance candidates toward clinic |
| Best fit for IND-bound programs |
| Target scope Single target |
| Problem addressed Refine potency and specificity |
| Data generated In vitro, In vivo and IND-supporting |
| Timeframe 18 months or less |
| Decision enabled Advance toward IND |
| DeliverableClinical-ready editors |
| Pipeline Model |
|---|
| Primary purpose Build and scale pipelines |
| Best fit for Multi-asset organizations |
| Target scope Multiple targets |
| Problem addressed Portfolio scale and differentiation |
| Data generated Program- and portfolio-level |
| Timeframe Varies based on scope |
| Decision enabled Pipeline-level commitment |
| Deliverable Clinical-ready editors for multiple targets |
Proof of Concept: Demonstrate That the Edit Works
The Proof-of-Concept model is designed to answer a foundational development question: can the right gene edit be made for this target? This work is typically completed in less than 6 months, providing rapid clarity to inform next steps. Engagements are structured to integrate with your scientific team, allowing collaboration at the level that best supports your internal strategy.
This path delivers feasibility for a single disease target. It is intentionally focused, rapid, and self-contained, allowing teams to generate meaningful specificity and efficacy evidence without requiring further commitment.
What this path enables
- Validation that a specific edit can be achieved at a defined locus
- Comparative assessment of multiple editing approaches, if desired
- Early confidence to advance—or stop—based on data
What’s included
- Genomic analysis to identify viable editing strategies
- Design and evaluation of a selection of editors across relevant modalities
- Cell-based validation with initial specificity assessment
- Delivery of 1–3 editors and supporting data to enable internal evaluation
Why teams choose this model
- Uncertainty of editing accessibility for a gene target
- Platform or modality evaluation
- Clear go/no-go decisions before downstream investment
Proof of Concept Model
Single target Feasibility and initial safety 1-3 editors for your target
Stop or move to Clinical Candidate Model
3 to 6 months
Clinical Candidate Model: Advance Toward the Clinic with Confidence
Whether you demonstrated proof-of-concept with us or have your own existing program, the clinical candidate model is built for programs to move beyond feasibility – and now are ready to rapidly translate promising edits into clinical-ready assets. Development proceeds in close collaboration with the sponsor R&D team, with shared oversight on design, candidate selection, and in vivo validation. ElevateBio’s role is scoped to compliment internal capabilities and align with program needs.
This path focuses on a single target and delivers optimized candidates supported by in vivo data and robust IND-relevant datasets. It is designed to reduce translational risk and support confident advancement toward regulatory milestones.
What this path enables
- Selection of optimized gene editing candidates for clinical application
- Generation of in vivo and supporting datasets
- Alignment with regulatory and clinical development expectations
What’s included
- Down-selection of editing approach and optimization of potency and specificity for your gene locus
- in vitro and in vivo evaluation in relevant models
- Safety, off-target, and performance characterization
- Data packages suitable for IND planning and regulatory interaction
Why teams choose this model
- IND-bound programs
- Transition from discovery to development
- Need for rigorous, decision-enabling data
Clinical Candidate Model
Proof of concept: Single target feasibility
Refine potency and specificity
in vitro & in vivo, Generate
IND-supporting data
3 to 6 months
This model is designed to move therapeutic programs forward with evidence to support decision-making on the path to the clinic. Whether you get here through our proof-of-concept model or independently, we are here as your preferred partner to help advance your program.
Pipeline Model: Build a Differentiated Gene Editing Pipeline
The pipeline model is designed for organizations building multiple genetic medicine programs that require sustained access to advanced gene editing innovation. Through this model, partners gain access to ElevateBio’s broad spectrum of gene editing modalities, including a portfolio of patented editing tools, and improved tailor-made editing systems with your edit in mind, enabled by our proprietary protein discovery engine powered by generative AI, to support pipeline development across selected targets.
This approach enables long-term differentiation by combining exclusive target access with ongoing discovery, rather than one-time delivery of individual assets.
What this path enables
- Pipeline development across multiple disease targets
- Continuous discovery and evolution of editing solutions
What’s included
- Access to ElevateBio’s full spectrum of gene editing modalities
- AI-enabled discovery and design of novel and highly potent editing compositions
- Application of multiple editing modalities across defined targets
- Collaborative development with ElevateBio scientists
Why teams choose this model
- Multi-asset or platform-driven strategies
- Need for sustained access to ElevateBio tools and scientists
- Desire to differentiate in competitive disease areas
- Time and cost synergies with multiple targets
Pipeline Model
Multiple targets: Feasibility & initial safety data Iterative development
Access to Editing Toolbox & Pipeline-Level Commitment
Timeline varies by agreement
Explore a strategic partnership
Partner engagements provide access to selected targets, ElevateBio’s gene editing tools, and discovery capabilities.
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