🧬Genetic therapies, NACFC Highlights recording, care survey, + more!


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CF RESEARCH YEAR-END UPDATE

The Cystic Fibrosis Foundation is excited to share the latest news and developments in CF research. We are continuing to invest in genetic therapies that promise to - one day - offer a treatment for the underlying cause of cystic fibrosis in all people with the disease, including those with two copies of rare and nonsense mutations.

# Genetic Therapy Clinical Trials for CF


SpliSense ([link removed])
completed a Phase 1 clinical trial of its inhaled anti-sense oligo drug. This therapy is designed to correct splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in people with the CF-causing mutation, 3849+10kb. The trial showed the drug was safe and well tolerated in people with this splicing mutation. A Phase 2 clinical trial testing the efficacy of the drug is being planned for the first half of 2024.

In November 2023,
4D Molecular Therapeutics ([link removed])
(4DMT) reported promising interim results from an
ongoing Phase 1/2 clinical trial ([link removed])
of its gene delivery technology -- an inhaled adeno-associated virus used to transport a non-mutated copy of CFTR DNA into lung cells. The results indicated safety and provided evidence of delivery and expression of the delivered copy of CFTR in lung tissue samples of participants. We are investing up to an additional $2.8 million in 4DMT to support preclinical discovery and development of other adeno-associated viruses for treatment of CF, bringing our total support for the additional program up to $6.6 million.



# Investing in Genetic Therapies

We are investing up to $3 million in
ViaNautis ([link removed])
to conduct discovery stage research focused on the development of a non-viral gene delivery technology that could carry different genetic therapies -- such as CFTR messenger RNA (mRNA), CFTR DNA, or potentially gene editing machinery -- to targeted cells in the CF lungs.

We also are investing up to an additional $9 million in
Arcturus Therapeutics ([link removed])
to conduct clinical trials of its inhaled
mRNA therapy ([link removed]). The funding brings our total commitment to Arcturus to approximately $25 million. Arcturus has completed an initial safety study in 32 healthy volunteers and has now dosed the first person with CF in their Phase 1b trial. Both studies are being conducted in New Zealand. If successful, the genetic-based therapy would treat the underlying cause of CF by providing the correct RNA template necessary for lung cells to produce a functional CFTR protein.



# To learn more about the different types of genetic therapies and how they could work:

* Take a look at our
genetic therapies section ([link removed])
on our website.
* Download our
Understanding Genetic Therapies ([link removed])
infographic.
* Find genetic therapy clinical trials that are currently enrolling patients on the
Clinical Trial Finder ([link removed]).
* Browse the
Drug Development Pipeline ([link removed])
to see some of the genetic therapy research we're funding.





# In Case You Missed It

The
recording of the December 5 NACFC Highlights Webinar ([link removed])
is available. Watch for the latest research updates in genetic therapies, mental health, and other topics.



# Have Thoughts on CF Care?

We need to hear from you. Share your thoughts on how the medical care received at CF Foundation care centers can continue to meet your or your loved ones' needs.
Take the Adapting CF Care survey ([link removed])
by December 20. Highlights from survey results will be available in early 2024.



# Connect With Others at BreatheCon

Adults with cystic fibrosis are invited to join their peers February 9-10 for
BreatheCon ([link removed]). Through small group discussions, you'll have the opportunity to discuss life without modulators, the complexities of aging with CF, the twists and turns of managing CF-related diabetes, and so much more.
Register today ([link removed])!
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Drug Development Pipeline ([link removed])
•
Clinical Trial Finder ([link removed])



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www.cff.org ([link removed])


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