In gene editing news, Tome Biosciences revealed its plans to use its technology to treat a rare disease called phenylketonuria and to design a natural killer cell therapy for autoimmune diseases. A Phase 1 trial using CRISPR to remove HIV from patient genomes was unsuccessful, but showed potential for future combination treatments. Maze Therapeutics has partnered with Shionogi to develop a Pompe disease treatment after a previous deal with Sanofi was blocked by the FTC. Lastly, Pfizer and AstraZeneca announced new investments of nearly $1 billion in France.
Key takeaways:
- AC Immune has sold global licensing rights to an experimental immunotherapy for Alzheimer’s disease to Takeda, receiving $100 million and potentially another $2.1 billion in future payments.
- Artificial Intelligence (AI) has not yet proven to be effective in speeding up the development of n-of-1 treatments for rare genetic diseases, according to a new study.
- The FDA has been criticized for not providing clear guidelines on how to list patents for drug-and-device combination products in the Orange Book, leading to frustration among industry players and patient advocates.
- A Phase 1 trial using CRISPR editing to remove HIV from patient genomes was unsuccessful, but one patient's infection did not rebound for about four months after stopping antivirals, suggesting that CRISPR editing might be used in combination with other approaches to cure HIV in the future.