CGT 1>8

Cell and gene Therapies 1>8 in preparation with the aim of achieving highly efficient and affordable solutions.

Family

Description

Examples

8 Major Families of Cell and Gene Therapies

Family

Description

Examples

Gene Editing Therapies

Techniques that modify an organism's genetic material

CRISPR-Cas9, TALENs

Gene Replacement Therapies

Introducing a normal copy of a gene to compensate for a mutated one

Luxturna (for Leber congenital amaurosis)

Gene Silencing Therapies

Inhibiting or silencing expression of dysfunctional genes

Antisense oligonucleotides, siRNA

Viral Vector Therapies

Using viruses to deliver therapeutic genes into cells

AAV (adeno-associated virus), lentiviral vectors

Stem Cell Therapies

Using stem cells to regenerate damaged tissues or produce healthy cells

Hematopoietic stem cell transplant

CAR T-Cell Therapies

Genetically modifying T-cells to target specific cancer cells

Kymriah, Yescarta

Protein Replacement Therapies

Administering proteins to compensate for deficient or absent ones

Enzyme replacement therapies

Oncolytic Virus Therapies

Using viruses that selectively infect and kill cancer cells

Talimogene laherparepvec (T-VEC)

Considerations accompanying Classification

1. Mechanism of Action: Therapies can act through multiple pathways; for example, gene-editing techniques can both replace and silence genes.

2. Combination Therapies: Some therapies may combine elements from multiple families; for instance, CAR T-cell therapies can involve both gene editing and viral vector delivery.

3. Evolving Technologies: The field is rapidly advancing, so new methodological approaches could emerge, leading to the creation of subfamilies or subcategories.

This classification framework can help facilitate understanding, research, and clinical application of cell and gene therapies, while acknowledging the complexities inherent in their development and implementation.

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