Recent Developments in Biotechnology: CRISPR

Private equity and venture capital firms also are banking on CRISPR-based therapeutics’ high growth potential. Frost & Sullivan found that overall investment in the space has already crossed $1 billion—a value expected to increase further over the next 3 to 4 years. Here are some important initiatives in this field.

In a recent article, Frost & Sullivan examined the potential of CRISPR/Cas9 technology to slow the progress of hearing loss, improve algal yields and combat the spread of mosquito-borne diseases. But so much research is being conducted in this area that further exploration is warranted.

CRISPR refers to clustered regularly interspaced short palindromic repeats, and Cas9 is a CRISPR-associated endonuclease. CRISPR/Cas9 is a genome-editing technique in which a short RNA guides a DNA-cutting Cas9 enzyme to a precise, targeted location in the genome to selectively delete, modify or correct a disease-causing dysfunction in that specific DNA segment. Compared with older gene-editing techniques, including zinc-finger nuclease (ZFN) and transcription activator-like effector nuclease (TALEN), CRISPR/Cas9 has proven to be cheaper, more accurate and more efficient. CRISPR is almost 150 times less expensive than the ZFN method, costing as little as $30 in high-volume requirements. Because there is no domain and nuclease fusion, it supports multiplex targeting.

Since its inception, CRISPR has gained substantial traction in the global research community because of its vast, disruptive potential. Key growth opportunities include the development of new therapeutics applications, end-to-end research tools, and reagent offerings; drug compound discovery; the development of agriculture-focused tools; and innovations in animal and disease models for drug discovery.

Private equity and venture capital firms also are banking on CRISPR-based therapeutics’ high growth potential. Frost & Sullivan found that overall investment in the space has already crossed $1 billion—a value expected to increase further over the next 3 to 4 years. Pharmaceutical companies also are financially supporting these firms by signing product development-focused partnerships. Funding from governmental agencies, such as the National Institutes of Health, also has increased multifold. Frost & Sullivan continues to identify important initiatives in this field.

Broad Institute, (Cambridge, Mass.)

Scientists at the Broad Institute have developed a CRISPR-based tool for virus detection that they named SHERLOCK, an acronym for Specific High-sensitivity Enzymatic Reporter unLOCKing. The highly sensitive detector can find even a single molecule of RNA or DNA in the target sample. The tool can be developed as a paper-based test, with significantly higher sensitivity than the most commonly used protein diagnostic technique: enzyme-linked immunosorbent assay (ELISA). Demonstrated applications of this tool include detecting the presence of the Zika virus in blood or urine samples, detecting antibiotic-resistant genes, and identifying cancerous mutations.

Thermo Fisher Scientific (Waltham, Mass.)

Thermo Fisher Scientific has one of the most comprehensive CRISPR research tools and reagent portfolios. Leading industry resources have already recognized its Invitrogen LentiArray CRISPR libraries as the most comprehensive and game-changing products.  The company has signed commercial and product development agreements with leading pharmaceutical companies, including AstraZeneca.

Dharmacon, a Horizon Discovery Group Co., (Lafayette, Colo.)

Dharmacon offers a comprehensive set of CRISPR/Cas9 gene-editing tools. Horizon Discovery acquired Dharmacon in September 2017 for $85 million. It is a leader in RNA interference (RNAi) products with a strong portfolio of CRISPR reagents and arrayed libraries. Niche product offerings include predesigned sgRNA, vector-based and DNA-free Cas9 nuclease, custom guide RNA design and ordering tools, and homology-directed repair (HDR) donor template design and ordering tools and kits.

CRISPR Therapeutics (Basel, Switzerland)

CRISPR Therapeutics has partnered with Bayer and Vertex Pharmaceuticals to develop CRISPR-based therapeutics for specific diseases. Other partners include Massachusetts Institute of Technology, Massachusetts General Hospital Cancer Center, StrideBio, MaxCyte and MaSTherCell. The company expects clinical trials for its first developmental product, CTX001, to start in Europe by the end of 2018. CTX001 would use CRISPR technology to modify DNA of the blood stem cells of patients outside the body. The engineered cells would then be reintroduced into the patients to treat disorders that affect oxygen transport in the body. The company has had significant growth in its immuno-oncology product portfolio.

Intellia Therapeutics (Cambridge, Mass.)

Frost & Sullivan identified Intellia Therapeutics as one of the most promising start-ups in the CRISPR-based therapeutics segment. Leading pharmaceutical companies Novartis and Regeneron have backed the company, whose key therapeutic focus areas include immuno-oncology, hematopoietic stem cells, and liver diseases. Considering Intellia's strong R&D pipeline, Frost & Sullivan expects the company to become a leader in this segment.

The Road Ahead

While gene editing technologies affect animal and plant genetic engineering, the area witnessing the most disruption is expected to be human cell line engineering to develop of new drugs.  CRISPR, with its broad applications in health care and its cost-effectiveness, has eclipsed other gene editing technologies.

With low barriers to entry/exit in the fragmented CRISPR research tools market, Frost & Sullivan expects significant merger and acquisition activity over the next 3 to 5 years as large companies look to strengthen their product and technology portfolios and increase their market shares, and as companies operating in human therapeutic applications try to expand or enhance their product portfolios to satisfy researchers who typically prefer a single vendor to ensure compatibility across products and tools.

With its value already proven in research, the next breakthrough will be to establish CRISPR as a safe and effective treatment. It may one day be widely acceptable to design or alter genes in animals, plants, and humans to irreversibly alter the gene pool and the course of evolution. CRISPR isn’t a costly or unobtainable technology: it’s accessible and in use now.

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