Our latest developments


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RESEARCH + CARE

# April Update

Here are the latest news and developments in cystic fibrosis research and care from the Cystic Fibrosis Foundation:

Genetic Therapies

# Investing in Potential Splicing Mutation Treatment

We are investing up to $8.5 million in additional funds in
SpliSense ([link removed])
to support a planned Phase 2 clinical trial of its inhaled
antisense oligonucleotide (ASO) ([link removed])
drug for people with CF who have the splicing mutation 3849+10Kb C-to-T. The objective of the clinical trial is to generate a proof-of-concept for using this type of technology to treat people with CF with certain rare mutations.

# ReCode Therapeutics mRNA Therapy

In February,
ReCode Therapeutics ([link removed])
dosed the first participants of a Phase 1 trial of an inhaled
messenger RNA therapy ([link removed])
(mRNA), which could provide a treatment option for all people with CF, regardless of their mutations. ReCode's mRNA therapy is designed to provide a correct copy of the mRNA to lung cells to make a functional CFTR protein.

# Genetic Therapies Webinar Recording Now Available


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In case you missed it, the
recording from our genetic therapies research webinar is available in English ([link removed])
and
Spanish ([link removed]). Starting from the basics, learn about genetic therapies in CF, including how they differ from existing therapies and the benefits they can potentially provide to any person with CF, including people with nonsense and rare mutations. Katie Howe, one of the featured speakers, is the Foundation's new clinical trial navigator -- a designated individual who provides personalized, one-on-one assistance to people with CF and their families who have questions about clinical trials. Contact Katie at [email protected] link in text (mailto:[email protected]) or by
filling out an inquiry form ([link removed]).

CFTR Modulator News

We invested more than $6.6 million in additional funding in
Sionna Therapeutics ([link removed])
to continue research into the development of potential new modulator therapies that could provide alternatives for people with CF who can't tolerate current modulators.

Infection Research

We provided an additional $5 million in
BiomX Inc. ([link removed])
to support a Phase 2b clinical trial to test the efficacy of bacteriophage (phage) therapy in treating chronic
Pseudomonas aeruginosa ([link removed])
infections -- the second most common type of lung infection in people living with CF. Phage therapy uses phages (bacteria-specific viruses) to kill specific bacterial strains that are causing an infection.

New CRMS Guidelines

The Foundation has published
new guidelines for CFTR-related metabolic syndrome (CRMS) ([link removed]), a classification that describes someone whose newborn screen indicated they might have CF, but their sweat chloride test results fell into an uncertain or borderline range. These new guidelines reflect increased knowledge of CRMS since the publication of the original guidelines in 2009 and contain recommendations for CF specialists and primary care providers about the care of people with CRMS, specifically genetic testing and counseling; monitoring, testing, and treatment; and communication with people with CRMS and families.

Share Your Thoughts About the Cost of CF

How has the cost of living with cystic fibrosis affected your daily life and access to healthcare services? Adults with CF and caregivers of people with CF who are U.S. residents are invited to
participate in our research survey ([link removed])
and help the Foundation understand how to inform and support the CF community and care teams in addressing financial issues associated with CF.

Take the survey today >> ([link removed])


ResearchCon


Join us at ResearchCon April 30 – May 1 ([link removed])
to hear what's new in CF science, research, and care. Engage with those living with CF and researchers who are studying the disease while hearing the latest updates on the drug development pipeline, evolving infection research, the CF Foundation Therapeutics Lab, and more.

REGISTER FOR RESEARCHCON

Educational Video on Health Equity in CF


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Every person with CF deserves access to a timely diagnosis, quality care, and community support. The CF Foundation created
an educational video ([link removed])
featuring Jennifer Taylor-Cousar, MD, and Tanisha Cunningham, an adult with CF and parent of a child with CF, to illustrate the importance of health equity to the CF community. Together, we can make a difference in the lives of people with CF -- improving care, research, and community support so that everyone benefits.

Watch the video >> ([link removed])


Other News

* In February, the Foundation published the results of a study in the
Journal of Cystic Fibrosis ([link removed])
on which rare mutations responded to elexacaftor/tezacaftor/ivacaftor (the components of Trikafta®) in the lab.
*
Anagram Therapeutics ([link removed])
is conducting a
Phase 1 clinical trial ([link removed])
of its potential enzyme therapy.
* April is National Donate Life Month, which aims to increase awareness about eye, tissue, and organ donation and
transplantation ([link removed])
and to encourage people to
register to donate their organs ([link removed]).





Stay connected with the CF Foundation via our social media channels or visit
cff.org/news ([link removed])
for the latest updates.


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