Gene Therapy Pipeline

How does it work

What is Gene Therapy

Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Gene therapies can work by several mechanisms: Replacing a disease-causing gene with a healthy copy of the gene, inactivating a disease-causing gene that is not functioning properly, and introducing a new or modified gene into the body to help treat disease. Gene therapy products are being studied to treat diseases including cancer, genetic diseases (like hearing loss and deafness), and infectious diseases.

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Gene Therapy products are becoming medically and commercially viable.

Gene-editing tools like CRISPR / Cas9 are making the development of gene therapy products realistic, efficient, and affordable.

30% to 50% of all hearing loss is due to genetic factors and medical providers are increasing their use of genetic testing as a reliable, realistic, cost-effective tool to identify causative genetic mutations.

Regulatory agencies like FDA and EMA are becoming more familiar with gene therapy technologies increasing the efficiency of the drug approval process.

Investment in Gene and Cell therapy companies is trending upwards – Private Institutional investment (VC and PE firms) in gene and cell therapy companies grew from $362M in 2020 to $68 Billion in 2021 (nearly 33% of all life science investment).

2023

Status Of RHI Product Line