Context: Bharat Biotech has launched NucelionTherapeutics, a wholly-owned subsidiary, marking a strategic expansion into the field of cell and gene therapies.
What is Nucelion Therapeutics?
- Type: It is a Contract Research Development and Manufacturing Organisation (CRDMO).
- Mandate: To provide high-quality, scalable process development and manufacturing solutions for advanced therapies to global life science innovators.
Gene Therapy: Core Concepts
- Definition: A medical technique that alters genetic material (DNA) to treat or prevent the root cause of a disease.
- Mechanism: Typically involves introducing a functional gene to compensate for a defective (mutated) one.
- Approaches: Gene replacement, gene inactivation (silencing), or introducing a new gene.
II. Key Classifications
1. Somatic Therapy:
- Targets body (somatic) cells.
- Changes are non-heritable (not passed to offspring).
- This is the standard, ethically accepted form of therapy.
2. Germline Therapy:
- Targets sex (germ) cells (eggs or sperm).
- Changes are heritable (passed to future generations).
- Widely banned due to major ethical concerns.
Delivery Methods:
1. Ex Vivo (Outside): Cells (like T-cells) are removed, modified in a lab, and re-infused. (e.g., CAR-T).
2 In Vivo (Inside): The gene is delivered directly into the patient’s body.
Tools and Vectors
- Vectors (Carriers): Used to deliver the new gene into cells.
- Viral Vectors: Modified, harmless viruses (e.t., Adeno-associated virus / AAV).
- Non-Viral Vectors: Synthetic systems (e.g., Lipid Nanoparticles / LNPs).
- Gene Editing Tools: Technologies that precisely modify existing DNA.
- CRISPR-Cas9: The most common tool for “cutting” and editing DNA.
- Base Editors: Newer, high-precision tools that can change a single DNA “letter” without cutting.
Major Applications:
- Monogenic (Single-Gene) Disorders:
- Sickle Cell Anemia, Hemophilia, ADA-SCID (Severe Combined Immunodeficiency), Muscular Dystrophy.
- Cancer:
- CAR-T Therapy: An ex vivo therapy that engineers a patient’s own T-cells (immune cells) to find and destroy cancer cells.
