Gene Editing for Rare Diseases
Identifying breakthrough, in-body CRISPR editing platforms to overcome the hurdles of first-generation technologies and deliver single-dose cures for monogenic diseases.
Client
Top-10 Biopharma Firm
Objective
Scout Next-Gen CRISPR Tech
Timeline
10-Week Sprint
Key Outcome
12 High-Priority Recommendations
The Challenge: Hurdles in Clinical Translation
CRISPR-based gene editing holds the promise of single-dose cures for thousands of monogenic diseases. While the therapeutic elegance is clear, clinical translation remains constrained by four interlocking hurdles that prevent widespread application beyond costly and complex ex vivo methods.
In-vivo Delivery
Safely shuttling large Cas nuclease complexes into hard-to-reach tissues like the CNS, retina, or bone marrow with high efficiency.
Off-Target Specificity
Avoiding unintended cuts or base conversions that could provoke oncogenesis or other deleterious mutations.
Immunogenicity
Balancing immune responses to foreign bacterial proteins (Cas) or delivery vehicles like viral capsids and LNPs.
Safety & Regulation
Demonstrating durable, lineage-stable edits and generating multi-year data to satisfy global regulators.
Our Approach: A Rigorous Discovery Framework
The client engaged August Research to uncover cutting-edge, clinic-ready technologies that go beyond first-generation CRISPR trials. We distilled the investigation into eight guiding questions and built a comparative matrix to evaluate solutions across four key axes co-defined with the client.
Evaluation Axes & Weightings
- 30% Delivery Efficiency
- 25% Safety Profile
- 25% Manufacturability
- 20% IP & Regulatory Clarity
Execution Timeline
Weeks 1-2: Baseline Landscaping
Mined >4,000 patents and 1,200 papers to shortlist 55 distinct technology leads.
Weeks 3-6: Deep-Dive Dossiers
Conducted 18 SME interviews and generated comparative safety/efficacy scorecards.
Weeks 7-8: Interim Review
Held a client workshop to re-prioritize to 22 solutions focused on CNS and hematology.
Weeks 9-10: Final Synthesis & Ranking
Delivered a heat-map dashboard and full diligence packs for the top 12 candidates.
Explore More of Our Work
See how our methodology delivers clarity and accelerates innovation across a range of complex technological challenges.