RSS Amplifier

Deep into the Forest · May 16, 2025

A Roundup of Interesting Therapeutic Advances

0
Sign in to vote or save

Bharath Ramsundar · Deep into the Forest

We discuss some interesting advances in therapeutics this week as well as providing a quick tour of some interesting science from across the web.

There has been some fascinating progress in the field of inverse vaccine design, which seeks to suppress specific parts of the immune response induced in autoimmune diseases. The field is in an early state but holds out the promising glimmer of a general strategy to retrain the immune system to tolerate rejected substances. These approaches have found some early clinical validation, but are probably several years out from mainstream adoption at the earliest. See this review article for an overview.

The New York Times has a noteworthy story about a customized patient-specific CRISPR treatment for a newborn baby with CPS1 deficiency, a rare disease affecting one in 1.3 million babies (see additional commentary from Nature). The treatment combined a lipid nanoparticle delivery mechanism with CRISPR editing to treat baby KJ. Thankfully, KJ appears healthy and growing normally after three rounds of treatment, but longitudinal tracking will be required to see the efficacy and safety of the treatment over the long term.

Deep Into the Forest is a newsletter by Deep Forest Sciences, Inc. Get in touch with us at partnerships@deepforestsci.com.

Author: Bharath Ramsundar, Ph.D.

Editor: Sandya Subramanian, Ph.D.

No posts

Read the original on deepforest.substack.com

Comments

Nothing yet. Say the first thing.

    Sign in to join the conversation.