Including genetic therapy investments, phage therapy highlights, and more


Cystic Fibrosis Foundation Standard Logo

CF RESEARCH UPDATE

Spring 2023


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The Cystic Fibrosis Foundation is excited to share the latest news and developments in CF research.

Genetic Therapy Investments

The Foundation is dedicated to advancing transformative treatments, such as genetic therapies, that could help everyone with CF, including those with rare and nonsense mutations. Genetic therapies aim to provide cells with the correct instructions for making the cystic fibrosis transmembrane conductance regulator (CFTR) protein.

We recently announced investments in three companies that are conducting lab research on different ways to deliver genetic therapies to the lung cells of people with CF.

* Up to $15 million to
ReCode Therapeutics ([link removed])
to further develop a unique, selective organ-targeting lipid nanoparticle to deliver healthy messenger RNA (mRNA) to lung cells.

* Up to $3.5 million to
Gensaic ([link removed])
to explore how to engineer bacteriophage or phage — specialized viruses that typically target bacteria — to deliver a healthy CFTR gene to lung cells.

* Up to $2 million to
Nanite Inc. ([link removed])
to study the development of polymer nanoparticles — novel delivery vehicles that can be programmed to target lung cells and resist the thick, sticky mucus that clogs the lungs of people with CF. The nanoparticles would initially be used to deliver mRNA therapy.

To learn more about the different types of genetic therapies and how they could work, take a look at our new and improved
genetic therapies section ([link removed])
on our website. We also created a handout called
Understanding Genetic Therapies ([link removed])
that you may find helpful.



Phage Therapy Spotlight

Since the
Infection Research Initiative ([link removed])
launched almost five years ago, we have funded more than $19 million in research — both lab work and clinical trials — into phage therapy, an experimental treatment that uses specialized viruses to kill specific bacterial strains.

Two companies that we have funded,
Armata Pharmaceuticals ([link removed])
and
BiomX Inc. ([link removed]), recently released some data from early-stage clinical trials using phage therapy to target chronic
Pseudomonas ([link removed])
infections in people with CF. Each company reported that their phage therapies were well tolerated and reduced the amount of bacteria in the lungs of people with CF who received the drugs. Armata is planning a Phase 2b study in people with CF. BiomX has already started the
second part of its Phase 1a/2b trial ([link removed])
and expects results later this year.

### Other Infection Research News

* Since achieving our $100 million funding goal last year, we have invested an additional $28 million to fight CF infections through the Infection Research Initiative.
*
Aridis Pharmaceuticals ([link removed])
reported positive results in a Phase 2a trial testing a non-traditional anti-infective, inhaled gallium, in treating Pseudomonas infections in people with CF. Inhaled gallium was found to be safe and well tolerated in people with CF who participated in the study. Aridis received FDA approval to continue the study using a higher dose. Results from that portion of the study are expected later this year.



### Other News

* Last week, we agreed to provide up to $15.5 million to
Anagram Therapeutics ([link removed])
to conduct early-stage clinical trials of a novel enzyme replacement therapy that could cut down on the number of pills most people with CF have to take to digest food properly. The company is planning to start a Phase 1 clinical trial over the summer.

* Don’t forget to
register to attend ResearchCon ([link removed]), a virtual CF community event taking place April 26-27. Learn and discuss the latest in CF-related science, research, clinical care, and lived experiences alongside members of the CF community, researchers, and clinicians.

* Insights from the CF community continue to influence the direction and conduct of CF research.
Join Community Voice ([link removed])
to offer your perspectives on upcoming research topics. For more information, read their
2022 Year in Review Report ([link removed]).





# Learn more about drugs in development and current studies:


DRUG DEVELOPMENT PIPELINE ([link removed])


CLINICAL TRIAL FINDER ([link removed])




4550 Montgomery Avenue, Suite 1100N | Bethesda, MD 20814 US


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