Regeneron buys 23andMe and has the ability to CHANGE DNA
Overview of Regeneron Pharmaceuticals
Regeneron Pharmaceuticals, Inc. is a leading American biotechnology company founded in 1988 and headquartered in Westchester County, New York. The company specializes in inventing, developing, and commercializing medicines for serious diseases across a range of therapeutic areas, including eye diseases, allergic and inflammatory conditions, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases5. Regeneron is recognized for its strong track record in translating scientific discoveries into approved treatments and innovative drug candidates5.
Regeneron's Technology Platforms
Key Technologies and Approaches:
- Antibody Engineering: Regeneron has developed proprietary technologies for rapid antibody discovery and development, which underpin many of its marketed drugs and pipeline candidates.
- Genomic Research: The Regeneron Genetics Center (RGC) is one of the largest and most diverse genomic databases in the world, enabling the identification of new drug targets and genetic drivers of disease. RGC uses advanced tools to mine the human genome for actionable insights, which are validated in preclinical models before advancing to human trials.
- Gene Editing and Gene Therapy: Regeneron is at the forefront of developing in vivo gene editing therapies, particularly through its collaboration with Intellia Therapeutics.
Regeneron and Changing Human DNA
CRISPR-Based Gene Editing
Regeneron has a significant partnership with Intellia Therapeutics to develop CRISPR/Cas9-based therapies. CRISPR/Cas9 is a revolutionary gene-editing technology that allows for precise modification of DNA at specific sites within the genome. This technology can:
- Knock out disease-causing genes: By inactivating or removing faulty genetic sequences, CRISPR can potentially cure or alleviate genetic diseases.
- Insert functional genes: Regeneron is pioneering approaches where a functional gene is inserted at a targeted site using CRISPR/Cas9 combined with adeno-associated viral (AAV) vectors, potentially providing a permanent cure for diseases caused by missing or defective genes.
Applications:
- Regeneron and Intellia have already demonstrated first-in-human gene editing with NTLA-2001 for transthyretin amyloidosis, showing significant reduction in disease biomarkers after a single dose.
- The companies are expanding efforts to target diseases beyond the liver, including neurological and muscular diseases, using Regeneron's proprietary antibody-targeted AAV vectors for precise delivery of gene-editing tools to specific tissues.
siRNA and Gene Silencing
Regeneron also collaborates with Alnylam Pharmaceuticals to develop small interfering RNA (siRNA) therapies. siRNA molecules can silence specific genes by degrading their messenger RNA (mRNA), thereby preventing the production of disease-causing proteins. This approach is being explored for diseases of the liver, central nervous system, and eye.
Potential Impact on Human DNA
Regeneron's technological advancements could change human DNA in the following ways:
- Permanent Correction of Genetic Disorders: CRISPR-based therapies can directly edit faulty genes in patients, offering the possibility of one-time, durable cures for inherited diseases.
- Targeted Gene Insertion: By combining CRISPR/Cas9 with viral vector delivery, Regeneron aims to insert healthy genes at precise locations, potentially treating conditions like hemophilia and Pompe disease.
- Gene Silencing: siRNA therapies can modulate gene expression without altering the underlying DNA sequence, providing another avenue for treating genetic diseases.
Summary Table: Regeneron's DNA-Altering Technologies
| Technology | Mechanism | Potential Applications | Collaboration Partners |
|---|---|---|---|
| CRISPR/Cas9 Gene Editing | Cut and modify DNA at target sites | Inactivate faulty genes, insert new genes | Intellia Therapeutics |
| Antibody-Targeted AAV Vectors | Deliver gene-editing tools to tissues | Targeted gene therapy beyond the liver | Intellia Therapeutics |
| siRNA Gene Silencing | Degrade specific mRNA | Silence disease-causing genes | Alnylam Pharmaceuticals |
| Genomic Data Mining | Identify new drug targets | Discover and validate novel genetic therapies | Internal (Regeneron Genetics Center) |
Conclusion
Regeneron Pharmaceuticals is leveraging advanced technologies-particularly CRISPR/Cas9 gene editing, targeted viral delivery systems, and siRNA gene silencing-to develop therapies that can directly modify or regulate human DNA. These innovations hold the promise of transforming the treatment of genetic diseases, potentially offering durable, one-time cures and fundamentally changing the landscape of medicine.
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