Panel, Conference Presentation
Where Is the Next Cure? | Global Conference 2024
Milken InstituteAlice Park, Noubar Afeyan, Aloe Blacc, Daphne Koller, Samarth Kulkarni, Pablo Legorreta
- New modalities including mRNA, CRISPR, and AI are projected to increasingly impact therapy and vaccines, potentially reducing drug discovery failure rates from the current 90–95% range, though small molecules and proteins are not expected to be fully replaced.
- CRISPR platforms are anticipated to reach regulatory approval approximately 10 years after elucidation, representing a significantly accelerated timeline compared to the historical 20–25 years, with in vivo editing for sickle cell disease potentially reaching price points of $10,000 to $20,000.
- AI is expected to transform every phase of drug discovery by identifying leads faster and disentangling biological complexity, though physical bench research remains a bottleneck; data collection is projected to surpass the sum of all historical data within the next three years.
- Manufacturing costs for allogeneic CAR-T therapies are expected to decrease by two orders of magnitude into the thousands of dollars range, with pig organ manufacturing expected to be inexpensive due to high litter sizes, eventually leading to global cost genericization.
- Royalty Pharma intends to continue ecosystem investments over the next 10 years, building on a track record of deploying $25 billion (60% in approved products and 40% in late-stage products), while research funding is expected to prioritize specific drug leads to prevent high-value trial failures.
- In the area of xenotransplantation, approximately 20 transplants of edited pig kidneys into decedent models are planned over the next 18 months with an initial goal of sustaining organs for two months, with human trials for compatible organ transplants expected within 18 to 24 months.
- By late this year or early next year, definitive phase three data for cancer vaccines is expected to emerge, with mRNA vaccines anticipated to show 40 to 50% improvements over Keytruda in melanoma, while approximately 9% of the U.S. population may benefit from GLP-1 drugs by 2030.
- The UK is expected to lead in proactive, data-centric healthcare regulation and disease data creation, potentially creating special regulatory pathways for rare diseases with strong patient narratives while requiring proof for broader-based indications.
- Preemptive medicine is expected to shift upstream to detect and intervene before disease manifestation, presenting challenges to current regulatory definitions of disease, while the underlying human biology is expected to be shared across populations despite the need for diverse genetic data.
- The field is approaching a societal tipping point regarding the balance between avoidable deaths and new risks, with expectations that healthcare focus on prioritizing human life may result in a slower economic slowdown during health crises, and that the social contract demands eventual cost reductions by orders of magnitude for global accessibility.