Latest News and Developments

Artificial Intelligence (AI)

Key Headlines: July 2025

• “AI Action Plan” Announced by the Trump Administration:
  • The White House has unveiled a major new policy — the “AI Action Plan” — designed to accelerate AI innovation and establish the U.S. as a leader in the global race for artificial intelligence dominance.
  • The primary pillars are:
    • Accelerating private-sector innovation
    • Expanding AI-related infrastructure
    • Promoting U.S.-based AI standards internationally
  • The plan embraces deregulation, aiming to reduce regulatory barriers, streamline federal oversight, and reward companies that produce AI systems the government deems “objective and devoid of ideological bias.”
  • Executive orders linked to this initiative are expected to be signed imminently, focusing also on modernizing the energy grid to support data center growth, including investment in nuclear power.
• Industry Implications:
  • The new approach stands in contrast with the previous administration’s focus on risk mitigation, instead enabling rapid AI deployment and reducing bureaucratic oversight.
  • The U.S. aims to challenge China’s advances in AI and position American technologies as the global standard.

Gene Therapy

Recent Breakthroughs & Regulatory Approvals

• CRISPR Therapy Advances:
  • Casgevy, the first CRISPR-based therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), has now been approved in several global markets and expanded to 50+ clinical sites.
  • Financing and access remain challenges, but reimbursement models based on outcome efficacy are being trialed in the U.K. and U.S.
  • The first personalized in vivo CRISPR treatment was delivered to an infant with a rare genetic disease within six months, establishing proof-of-concept for rapid, individualized gene editing.
  • In vivo genome editing research continues, promising safer, more accessible therapies versus current ex vivo approaches.
• Gene, Cell, & RNA Therapy Approvals:
  • As of Q1 2025, 33 gene therapies and 35 RNA therapies are approved globally with strong clinical pipelines.
  • Recent major approvals include:
    • Encelto (revakinagene taroretcel): First gene therapy for macular telangiectasia in the U.S.
    • QFITLIA (Alnylam’s siRNA therapy): Approved for hemophilia A/B in the U.S.
    • Afami-cel: First FDA-approved TCR-T therapy for metastatic/unresectable synovial sarcoma.
  • Oncology is now the dominant target for new gene therapy clinical trials.
• Industry Movement:
  • Eli Lilly acquired Verve for cardiovascular CRISPR therapies.
  • Kamau Therapeutics acquired IP from Graphite Bio for new SCD treatments.

Radioligand Theranostics

Precision Oncology and Market Expansion

• Landmark Approvals & Expanded Indications:
  • March 2025: FDA expanded approval of Pluvicto (177Lu; Lutetium-177-PSMA-617) for metastatic castration-resistant prostate cancer (mCRPC), based on phase 3 PSMAfore trial results showing a 59% reduction in disease progression or death risk.
  • The new indication triples the eligible patient pool by permitting earlier use after first-line ARPI and before chemotherapy.
  • Radioligand therapies are moving upstream in treatment and expected to become central in cancer care, thanks to tolerability and progression-free survival benefits.
• Industry Trends:
  • Significant pharma investments, including AstraZeneca’s $2B acquisition of Fusion Pharmaceuticals and Novartis’ $1B acquisition of Mariana Oncology assets.
  • Radioligand therapies now offer combined diagnostic and therapeutic capabilities (*theranostics*), enabling personalized treatments guided by precision imaging (e.g., PET selection for Pluvicto).
  • Advances in alpha-emitting isotopes (e.g., Ac-225) and manufacturing technologies drive innovation.

Notable Industry Moves

• Novartis’ Pluvicto achieved $1B sales in 2024, confirming commercial success and clinical demand.
• Major investments and pharma participation are accelerating theranostic and radiopharmaceutical pipeline expansion.

Recent Key Product Approvals (2024–2025)

Industry Outlook

Artificial intelligence, gene therapy, and radioligand theranostics are all experiencing simultaneous regulatory, commercial, and technological inflection points. The integration of personalized medicine, digital infrastructure, and increasing investment is driving faster innovation and expanding global patient access, particularly in oncology and rare diseases.

Key areas to watch include:

• In vivo genome editing clinical advances.
• Combination radioligand and immunotherapy clinical trials.
• Regulatory policy shifts from U.S. FDA and EMA that could impact global therapy approvals and market access.