Sun Sep 24 2023

Gene Therapy:

• Gene therapy uses viruses as medicines to treat genetic disorders.
• It has historically focused on single-gene disorders by providing missing genes or correcting mutations.
• Viruses are used for precise delivery of therapeutic genes to specific body locations.
• The field is evolving rapidly, with advancements in using viruses, bacteria, and cells as living medicines.
• Promising results have been seen in treating rare genetic disorders like Leber's congenital amaurosis.

Three Eras of Modern Medicine:

• The first era was chemical medicines synthesized from natural sources.
• The second era introduced protein medicines, such as enzyme treatments and antibody therapies.
• The current era focuses on living medicines, including viral gene therapies that use viruses to deliver therapeutic payloads.

Applications of Gene Therapy:

• Gene therapy can now be applied to broader indications like cancer treatment.
• Combining immunotherapy with viral gene therapy enhances cancer treatment effectiveness.
• Clinical trials show promise for brain cancer treatment using viral gene therapy in mice models.
• Other potential applications include reducing the need for sleep through DEC2 mutation correction and enhancing human abilities for survival on Mars.

FDA Hurdles and Aging as a Disease State:

• Regulatory hurdles cause delays in bringing new gene therapies to market due to extensive paperwork and FDA review processes.
• Expanding the FDA's definition of disease may allow regulatory approval of gene therapies aimed at enhancing human potential, including aging as a disease state.
• Accessing sufficient funding is a challenge for biotech companies before reaching significant milestones.

Biotech's Valley of Death:

• Early-stage biotech companies struggle to access sufficient capital before de-risking milestones.
• Scaling up manufacturing processes requires financial resources.

Human Enhancements:

• Gene therapy has the potential to enhance human abilities beyond treating diseases.
• Possibilities include night vision, rejuvenation treatments, altering genetic traits for survival in harsh environments, and mental health improvements through precise targeting of receptors or proteins in the brain.

Capital Markets Challenges:

• The biotech market has faced setbacks due to interest rate fluctuations.
• Lack of access to capital hinders progress in scaling up manufacturing capabilities.

Tool Chain for Single-point Edits:

• Advances have been made in developing tools for accurate single-point edits in DNA sequences, such as CRISPR.
• These techniques enable precise editing, correcting mutations or removing unwanted sections from the genome.