The mRNA-4157-P201 Moderna vaccine in combination with Merck’s KEYTRUDA®) effective against skin melanoma

Sensational results of a combination of Moderna’s mRNA vaccine with Merck’s KEYTRUDA in curing skin cancer. 

The next steps are to test mRNA vaccines to mitigate and perhaps heal pancreatic and other cancers. I am very optimistic that in a couple of decades from now, cancer will be curable.

A very big problem is the cost of developing these vaccines. The mRNA vaccines are created (personalised) for each specific individual depending on the cancer-causing mutations of that individual making it very costly to develop. Hopefully, it will be possible to standardise the vaccine and thereby lower the costs and make it more affordable.

 https://share.google/fXFT4cYPtC3BloLvx https://share.google/fXFT4cYPtC3BloLvx

A one-time administration of CRISPR-Cas9 modified gene permanently controls high cholesterol

 

https://time.com/article/2026/08/28/one-time-treatment-high-cholesterol-crispr-results/

https://www.nejm.org/doi/full/10.1056/NEJMoa2511778

• Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ANGPTL3

Authors: Luke J. Laffin, M.D., Stephen J. Nicholls, M.B., B.S., Ph.D., Russell S. Scott, M.B., Ch.B., Ph.D., Peter M. Clifton, M.B., B.S., Ph.D., John Baker, M.D., Ashish Sarraju, M.D. https://orcid.org/0000-0003-1649-2110, Shweta Singh, Ph.D., +6 , and Steven E. Nissen, M.D. https://orcid.org/0000-0002-7231-6464Author Info & Affiliations

Published November 8, 2025

N Engl J Med 2025;393:2119-2130

DOI: 10.1056/NEJMoa2511778

VOL. 393 NO. 21

Copyright © 2025

Abstract

Background

Angiopoietin-like protein 3 (ANGPTL3) inhibits lipoprotein and endothelial lipases. ANGPTL3 loss-of-function genetic variants are associated with decreased levels of low-density lipoprotein cholesterol and triglycerides and a decreased lifetime risk of atherosclerotic cardiovascular disease.

Methods

We conducted an ascending-dose phase 1 trial to assess the safety and efficacy of CTX310, a lipid-nanoparticle–encapsulated clustered regularly interspaced short palindromic repeats–Cas9 endonuclease (CRISPR-Cas9) messenger RNA (mRNA) and guide RNA targeting hepatic ANGPTL3 to induce a loss-of-function mutation. Adults who had uncontrolled hypercholesterolemia, hypertriglyceridemia, or mixed dyslipidemia and were receiving maximally tolerated lipid-lowering therapy received a single intravenous dose of CTX310 (0.1, 0.3, 0.6, 0.7, or 0.8 mg per kilogram of body weight). The primary end point was adverse events, including dose-limiting toxic effects.

Results

A total of 15 participants received CTX310 and had at least 60 days of follow-up. No dose-limiting toxic effects related to CTX310 occurred. Serious adverse events occurred in two participants (13%): one participant had a spinal disk herniation, and the other died suddenly 179 days after treatment with the 0.1-mg-per-kilogram dose. Infusion-related reactions were reported in three participants (20%), and one participant (7%) who had elevated levels of aminotransferases at baseline had a transient elevation in aminotransferases to between three times and five times as high as those at baseline, peaking on day 4 and returning to baseline by day 14. The mean percent change in ANGPTL3 level was 9.6% (range, −21.8 to 71.2) with the dose of 0.1 mg per kilogram, 9.4% (range, −25.0 to 63.9) with 0.3 mg per kilogram, −32.7% (range, −51.4 to −19.4) with 0.6 mg per kilogram, −79.7% (range, −86.8 to −72.5) with 0.7 mg per kilogram, and −73.2% (range, −89.0 to −66.9) with 0.8 mg per kilogram.

Conclusions

Editing of ANGPTL3 was associated with few adverse events and resulted in reductions from baseline in ANGPTL3 levels. (Funded by CRISPR Therapeutics; Australia New Zealand Clinical Trials Registry number, ACTRN12623000809639.)

Notes

This article was published on November 8, 2025, at NEJM.org.

A data sharing statement provided by the authors is available with the full text of this article at NEJM.org.

Supported by CRISPR Therapeutics.

Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.

CPRISPR Cas12a2 can shred DNA of cancer cells

Clinical trials could start in 2030. Click on the link below for details.

https://www.nature.com/articles/d41586-026-02268-z

Modification of epigenetic markers has wide implications to cure multiple diseases

 

"A handful of start-up firms are testing therapies that target specific epigenetic markers to treat everything from high cholesterol to a rare muscular disorder."

The first target FSHD, short for facioscapulohumeral muscular dystrophy may become curable as early as 2030.

The therapy does not use CRISPR CAS9 to slice the mutated genes but rather controls the epigenes that turn the genes on or off like a switch. Thus, the mutated disease causing genes can be turned off without risking slicing at the wrong place or unwanted rearrangement of the genes after having been sliced by CRISPR CAS9.

https://www.nature.com/articles/d41586-026-01976-w

Gene therapy for rejuvenation of damaged eye cells is being clinically tested on humans

World first clinical trial on humans to rejuvenate damaged eye cells using gene therapy have started after successful treatment on mice. Similar trials on damaged cells of other organs are a possibility in the future.

With so many different approaches being attempted to cure damaged i.e. mutated cells, it is only a matter of time until cancer becomes not only treatable but also curable.

 https://www.nature.com/articles/d41586-026-01836-7

NPE: Not Parent Expected (from FTDNA Blog)

Sometimes, genetic tests may bring unexpected results: not the parent your were expecting or not the surname you were expecting. The link below provides a very useful explanation including suggestions on how to react in such an eventuality.

https://blog.familytreedna.com/npe-non-paternal-event/?utm_source=FamilyTreeDNA&utm_campaign=c041e51948-RSS_EMAIL_CAMPAIGN&utm_medium=email&utm_term=0_b0f8841478-c041e51948-110480329

Molecular clock based on gene-activity data can predict time to death in humans

 Till now, a epigenetic clock based on methylation has been used to study ageing and the effect of diets and medication on ageing. A powerful new molecular clock based on gene-activity data can now predict biological ageing in rodents, monkeys and humans and can predict time to death in humans. This powerful tool could be very useful to study the impact of new drugs to slow ageing in humans.

https://www.nature.com/articles/d41586-026-01661-y

https://www.nature.com/articles/s41586-026-10542-3?utm_source=Live+Audience&utm_campaign=2f2a6b7480-nature-briefing-daily-20260527_COPY_01&utm_medium=email&utm_term=0_-33f35e09ea-51454576