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Big Ideas in 2024: Programming Medicine’s Final Frontier with Jorge Conde

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A16z predicts tech innovations for 2024, focusing on programmable medicines like gene therapy. FDA adopts aviation safety approach for efficient review of gene therapies. Approval of CRISPR therapy for genetic diseases marks significant advancement. Challenges include permanence, FDA regulations, and limited accessibility due to high costs. Builders aim to enhance biology engineering for scalable and affordable manufacturing. Video emphasizes potential of biology growth and upcoming topics in AI, maritime exploration, and voice-first apps.

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Predictions for Major Tech Innovations in 2024.
A16z forecasts innovations such as nuclear renaissance, AI in complex workflows, and democratization of miracle drugs like gp1s.
Over 40 big ideas for 2024 include smart energy grids and computer vision.
Partner Jorge K focuses on programming medicine's frontier, comparing biotech to reusable rockets like SpaceX.
FDA adopts aviation safety approach for therapeutic products, aiming for transparent and flexible processes to revolutionize medicine production and impact society positively.
Overview of Traditional Drug Development Process.
Drug development involves drug discovery, preclinical development, and clinical development stages.
Drug discovery focuses on finding a target in a disease and can take several years.
Preclinical development involves testing a molecule outside of humans to ensure it is safe and effective.
Clinical development includes human clinical trials in three phases, lasting around five to seven years.
Programmable medicine offers a transformative approach to drug development.
Gene therapy can be used to deliver genetic payloads to different cell types for various diseases.
Programmable medicines allow for the redirection or redeployment of components, increasing efficiency.
The process of clinical trials and regulatory approval can be significantly shortened with programmable medicines.
This new approach enables a more versatile drug development process.
FDA adapting to review gene therapies more efficiently.
Reusing components like lipid nanoparticles and AAVs to speed up the process while maintaining rigor.
Newly announced Office of Therapeutic Products reflects recognition of meaningful innovation in medicine.
FDA prioritizes safety and efficacy for patients while embracing advancements in gene therapy.
Agency's efforts indicate a shift towards a more adaptive and efficient review process for emerging therapies.
FDA partnering with drug development industry to innovate and expedite rare disease treatments.
Launching pilot program to speed up rare disease treatments, similar to Operation Warp Speed.
Approving cutting-edge gene editing medicines for clinical trials, showing a shift towards embracing new technologies.
First CRISPR therapy for sickle cell anemia beta thalassemia approved, a major advancement in genetic disease treatment.
Therapy involves editing cells outside the body and reintroducing them for a functional cure.
Approval of CRISPR as a medicine signifies a major milestone in the medical field.
The process of approval could become quicker and more efficient for future medications.
Programmable medicines utilizing CRISPR target diseases with known causes, presenting a new treatment approach.
This development has the potential to cure previously untreatable conditions, offering hope to patients.
The rapid progression from CRISPR's discovery to approval in just over a decade is considered remarkably fast.
Challenges and Blockers in Programmable Medicines
Permanent edits in DNA for programmable medicines can pose risks of toxicity and require high safety evaluation from the FDA.
Access to complex programmable medicines is difficult as they are not readily available over the counter, leading to a lengthy treatment process.
The approval process for CRISPR therapy for sickle cell anemia highlights the complexities involved in accessing and administering programmable medicines.
Advanced therapies face limitations in accessibility due to high costs and lengthy hospital stays.
Despite the expensive nature of these therapies, they offer life-saving benefits.
Builders can improve biology engineering to make manufacturing more scalable and cost-effective.
Enhancements in programmable medicines and precision interventions can address issues of permanence and toxicity.
The ultimate goal is to develop better applications at a larger scale and lower cost to reach patients more efficiently.
Biology on an Exponential Scale
All life originated from a single cell, showcasing potential for growth and advancement.
Emphasis on upcoming topics: new age of maritime exploration with AI, AI-first games, and voice-first apps.
Encouragement for viewers to explore a full list of 40+ big ideas on the website.