Good Day BIO: CRISPR's first decade – plus, HHS appropriations, monkeypox, and more

We’re celebrating the 10-year anniversary of the discovery of CRISPR Cas9—and what it’s done for humans, animals, and plants. While it’s almost the holiday weekend, there’s a lot happening in D.C.: House Appropriations is marking up HHS funding for fiscal year 2023, and the Biden administration is preparing for monkeypox vaccinations and fall COVID vaccinations. (601 words, 3 minutes)

CRISPR’s first decade is packed with innovation

Ten years ago this week, Jennifer Doudna and Emmanuelle Charpentier discovered how to use RNA to program CRISPR-Cas9 to precision edit DNA—and the possibilities for improving the health of humans, animals, and plants have been unfolding at a remarkable pace.

What is CRISPR? Short for “Clustered Regularly Interspaced Short Palindromic Repeat,” CRISPR describes repetitive DNA sequences that bacteria store naturally to “remember” the DNA of viruses that attack them. Cas9 is a natural mechanism by which bacteria defend themselves by cutting a piece of DNA from the virus.

On June 28, 2012, Jennifer Doudna and Emmanuelle Charpentier published their discovery in Science. They shared the Nobel Prize in 2020.

Watch: Nobel Laureate Dr. Jennifer Doudna explains CRISPR for plant breeding 

CRISPR-Cas9 unlocks a range of medical applications—especially in the exciting new field of gene therapy, with clinical trials underway for blood disorders, cancers, inherited eye disease, diabetes, infectious disease, inflammatory disease, and protein-folding disorders, according to the Innovative Genomics Institute.

Just a few examples include:

• Engineering CAR T cells to enable personalized immunotherapy.
• Treating a hereditary form of blindness.
• Addressing transthyretin amyloidosis, a rare and fatal condition attacking the heart and nerves.
• Eliminating sickle cell disease and beta thalassemia in several patients.

CRISPR-Cas9 can also improve the health of animals, and facilitate a One Health approach to animals, humans, and the environment. Examples include:

• Producing chickens resistant to bird flu and ticks that don’t carry as much disease.
• Better identifying salmonella varieties in chicken production.
• Producing heat-tolerant cattle with shorter hair—the first gene-edited food animal granted FDA’s “low-risk determination.”

And CRISPR-Cas9 can dramatically speed up plant breeding, which has led to developments like:

• Tomatoes with high vitamin D content.
• Strawberries with improved yield and longer shelf life.
• Potatoes less prone to browning.
• Drought-resistant wheat and tea.
• Tastier decaf coffee.

House Appropriations will mark up the FY23 HHS budget – here's what's in it

The House Appropriations Committee today will mark up the bill covering appropriations for Health and Human Services (among other agencies) for fiscal year 2023—and many of BIO’s requests have been included.

A few things to highlight in the draft legislation:

• $845 million ($100 million above FY22 enacted level) for Biomedical Advanced Research and Development Authority (BARDA)
• $855 million ($10 million above FY22) for the Strategic National Stockpile
• $800 million ($20 million above FY22) for Project BioShield
• $353.2 million ($100 million above FY22) for FY23-FY25 for CDC’s Global Health Protection
• $382 million ($117 million above FY22) for pandemic influenza
• $35 million ($15 million above FY22) for the CDC’s Advanced Molecular Detection program
• Report language (page 235) regarding antimicrobial resistance requesting a “professional judgement budget estimate to support a comprehensive, long-term program for the development and procurement of novel antibiotics.” 

Click here to read the full list of BIO’s requests for the year. 

The context: “The budget blueprint lays out post-pandemic priorities and reverses a decade-old ban on congressional earmarks, allowing lawmakers to target federal spending toward projects in their districts,” explains Axios.

More Health Care News:

White House: Biden-⁠Harris Administration’s monkeypox outbreak response
“The vaccine strategy will help immediately address the spread of the virus by providing vaccines across the country to individuals at high risk,” says the fact sheet, including the JYNNEOS vaccine developed by BIO member Bavarian Nordic and the ACAM2000 vaccine developed by BIO member Sanofi.

President Biden’s Thursday: Delivering final remarks to the three-day NATO conference. Meanwhile, the Biden administration has placed a $3.2 billion order for Pfizer’s updated COVID vaccine for the fall, reports The Hill. 

What’s Happening on Capitol Hill: The House Appropriations Committee will mark up the FY23 budget for several departments including Health and Human Services (HHS), Labor, and Education (see story above for details on BIO’s funding requests).