Search

Page Title

Drug Development Pipeline

We’re attacking CF from every angle.

Click on any drug in the interactive pipeline below to learn more about CF drugs in development or already in use by patients.

Click on any drug in the interactive pipeline below to learn more about CF drugs in development or already in use by patients.

Drug Descriptions

Genetic Therapy

  • SPL84

    This program is working to develop a potential therapy for people with CF who have splicing mutations. Splicing is an essential process in which RNA is cut into pieces and then stitched back together in a specific way. Splicing mutations in the CFTR gene cause the RNA to be cut or stitched incorrectly, leading to a mutated CFTR protein. SPL84 is a short nucleotide, or a small piece of genetic material, that is designed to bind to RNA and change its properties in specific ways. In the case of a splicing mutation, the short nucleotide is designed to ensure that the RNA is cut and stitched correctly, allowing functional CFTR protein to be made.

  • Eluforsen (QR-010)

    Eluforsen (QR-010) is an oligonucleotide designed to repair CFTR-encoded mRNA, which could result in a normal CFTR protein in people who have one or two copies of the F508del mutation. It is delivered via inhalation.

  • 4D-710

    This program is working to advance a gene delivery vehicle that targets cells in the lung. 4D-710 is a customized adeno-associated virus (AAV) vector designed to deliver a healthy CFTR gene specifically to cells in the lungs of people with CF. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation.

  • ARCT-032 (LUNAR®-CF)

    This program is developing a potential inhaled therapy to deliver normal CFTR messenger RNA (mRNA) to the lungs. Lung cells would then use the instructions in the mRNA to create functional CFTR protein. This type of therapy could work for any person with CF, regardless of their CFTR mutations.

  • RCT2100

    This program is studying messenger RNA (mRNA) delivery to treat rare CFTR mutations, including nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Delivery of mRNA would allow lung cells to make full-length, functional CFTR protein.

  • VX-522 mRNA

    VX-522 is an inhaled messenger RNA (mRNA) therapy. It aims to deliver a full-length copy of CFTR mRNA to lung cells using a lipid nanoparticle. Lung cells would then use the instructions in the mRNA to create functional CFTR protein. This type of therapy could work for any person with CF, regardless of their CFTR mutations.

  • MRT5005

    MRT5005 is a drug designed to restore CFTR function by delivering correct copies of CFTR-encoded mRNA to the lungs. mRNA is a molecule that contains genetic instructions to make proteins. Delivery of CFTR-encoded mRNA would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. This drug is delivered via inhalation.

  • Spirovant Sciences

    This program is working to advance inhaled adeno-associated virus (AAV) delivery of the CFTR gene to the lungs. This would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. 

  • Carbon Biosciences

    This program aims to deliver a functional CFTR gene directly into the lung cells of people with cystic fibrosis using a cutting-edge approach, allowing the lung cells to create normally functioning CFTR protein. This approach has the potential to provide a treatment for all people living with CF, regardless of mutation. 

  • Carmine Therapeutics

    This program is working to develop a non-viral method to deliver a healthy copy of the CFTR gene into the lung cells of people with CF. This would allow cells to create normally functioning CFTR protein, regardless of an individual's specific CFTR mutation.

    This program plans to use extracellular vesicles — bits of cell membrane that naturally bud off from cells to form tiny particles. Once loaded with the gene therapy, these particles can be either inhaled or infused through an IV. This method of delivering healthy CFTR genes shows promise because it harnesses a natural process in the body that is unlikely to trigger an immune system response.

  • Gensaic

    This program is developing a novel approach to deliver a gene therapy to the lung cells of people with CF. Researchers are exploring how to engineer bacteriophage — specialized viruses that typically target bacteria — to deliver the CFTR gene to people with CF. Researchers believe phage-derived particles might be effective because they are unlikely to trigger an immune response, allowing the gene therapy to be re-dosed when needed. Other advantages of this approach include the potential to target specific organs with full-length human genes.

    If successful, this gene delivery vehicle would deliver a healthy CFTR gene to lung cells, which would allow the cells to produce functional CFTR protein. This approach has the potential to provide a treatment for all people living with CF, regardless of mutation.

  • Nanite

    This program is exploring polymer nanoparticles, a type of delivery vehicle that could be used to carry genetic therapies into the lung. Compared to other delivery vehicles, these polymer nanoparticles could more accurately target lung cells and potentially better resist the thick, sticky mucus that clogs the lungs of people with CF.

    At first, Nanite will work on developing polymer nanoparticles that can deliver messenger RNA (mRNA) therapy into the lungs. Delivery of mRNA would allow lung cells to make full-length, functional CFTR protein, regardless of an individual’s CFTR mutations. Eventually, Nanite hopes these nanoparticles can be used to deliver other types of genetic therapies, such as gene therapy or gene editing, and target other organs affected by CF, such as the pancreas. 

  • Pioneering Medicines

    This program is a first-of-its-kind collaboration to spur the development of genetic-based therapies for cystic fibrosis. Through this agreement, Pioneering Medicines will combine technologies from several companies to develop potential treatments for CF.

    At the onset, the program will focus on developing technology to determine whether it can create a functional CFTR protein in lung cells, and a gene writing approach that may enable the simultaneous correction of numerous types of mutations in the CFTR gene. These two strategies will be combined with a delivery approach focused on targeting the proper cells in the lung and potentially other tissues.

  • Prime Medicine

    This program is developing an approach called prime editing, a type of gene editing that has the potential to correct many different types of CF-causing mutations. Prime editing involves inserting or deleting small pieces of DNA at precise locations in a gene.

    This program is studying two different technologies using use prime editing to fix the CFTR gene. The first, called “hotspot,” uses prime editing to make smaller corrections to specific CFTR mutations. Prime Medicine has already begun to use hotspot to correct the G542X nonsense CFTR mutation in the lab and will extend this work to develop therapies for other CFTR mutations. The second, called PASSIGETM, uses prime editing to make large gene insertions, which could potentially work in nearly any person with CF, regardless of their underlying CFTR mutations.

  • SalioGen Therapeutics

    This program is studying a novel Gene Coding™ approach that is designed to turn on, turn off, or modify the function of any gene in the genome. In CF, this approach seeks to insert a large piece of healthy CFTR DNA at a precise location within the CFTR gene, which could enable the expression of a functional CFTR protein in essentially all individuals with CF, regardless of their individual mutation.

  • ViaNautis Bio

    This program is developing a new method to deliver genetic therapies to the lungs of people with CF. polyNaut® is a non-viral genetic therapy delivery mechanism that aims to reach targeted cells in the CF lungs and carry large genetic therapy payloads — two major challenges in delivering a CF genetic therapy. This approach may also allow for redosing of the genetic therapy, a useful option if the therapy’s effects are not permanent.

Restore CFTR Protein

  • Elexacaftor + tezacaftor + ivacaftor (Trikafta®)

    Elexacaftor + tezacaftor + ivacaftor (Trikafta®) is a combination therapy combining three CFTR modulators. Elexacaftor and tezacaftor are CFTR correctors, a type of modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Ivacaftor is a potentiator. Once CFTR protein reaches the cell surface, potentiators help facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell. 

  • Ivacaftor (Kalydeco®)

    Ivacaftor (Kalydeco®) is an oral medication that was the first drug available that targeted the underlying cause of CF – the defective CFTR protein.  Ivacaftor helps facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. 

  • Lumacaftor + ivacaftor (Orkambi®)

    Lumacaftor and ivacaftor (Orkambi®) is a combination therapy combining lumacaftor, which is designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface, with ivacaftor, which helps improve the function of the protein as a chloride channel on the cell surface.

  • Tezacaftor + ivacaftor (Symdeko®)

    Tezacaftor + ivacaftor (Symdeko®) is a combination therapy combining tezacaftor, a compound designed to move the defective CFTR protein to the proper place in the airway cell surface, with ivacaftor, which helps facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell.

  • Vanzacaftor + tezacaftor + deutivacaftor

    This program is studying a combination therapy combining three CFTR modulators. Vanzacaftor (VX-121) and tezacaftor are CFTR correctors, a type of modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Deutivacaftor (VX-561) is an altered form of the potentiator ivacaftor (Kalydeco®). Potentiators are drugs that facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. Deutivacaftor may be more stable in the body than regular ivacaftor, which would allow it to be taken once a day.

  • Ataluren

    Ataluren (Translarna™) is a novel, small molecule compound designed to enable production of a full-length and functional CFTR protein in individuals with CF who have nonsense mutations. The compound aims to help the body override a premature signal to stop production of the CFTR protein. 

  • VX-659 + tezacaftor + ivacaftor

    This program tested VX-659 in combination with tezacaftor and ivacaftor. VX-659 and tezacaftor (VX-661) are CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.

  • QBW251

    QBW251 is a type of CFTR Modulator called a “potentiator”. Similar to the drug ivacaftor, this drug would help to facilitate the opening of the chloride channel on the cell surface. This compound is administered through an oral pill.

  • ELX-02

    ELX-02 is a compound designed to restore CFTR function in people with CF who have nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. ELX-02 is intended allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.

  • ABBV-2222 + ABBV-3067 + ABBV-576

    This program studyied a combination therapy combining three CFTR modulators, two correctors and a potentiator. Correctors are a type of modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, potentiators help facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.

  • Cavosonstat (N91115)

    Cavosonstat (N91115) is a compound that modulates the function of the defective CFTR protein and decreases inflammation in the lung. Cavosonstat is the first of a new class of compounds that increase levels of an important signaling molecule in the body, called S-nitrosoglutathione or GSNO. Levels of GSNO have been shown to be decreased in people with CF. These novel compounds have been shown to increase the amount of CFTR protein that reaches the cell surface and to stabilize CFTR protein so that its function can be improved. 

     

  • Deutivacaftor (VX-561)

    Deutivacaftor is an altered form of the potentiator ivacaftor (Kalydeco®). Potentiators are drugs that facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. Deutivacaftor, or deuterated ivacaftor, may be more stable in the body than regular ivacaftor, which would allow it to be taken once a day.

  • FDL169

    FDL169 is a CFTR corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.

  • Nesolicaftor (PTI-428)

    Nesolicaftor is a type of modulator called an amplifier. Amplifiers increase the amount of CFTR protein in the cell. This makes more CFTR protein available for other therapies, such correctors and potentiators, to work on.

  • Posenacaftor (PTI-801)

    Posenacaftor is a type of CFTR modulator called a corrector. Correctors are drugs designed to fix and restore the function of the defective CFTR protein. The corrected CFTR then moves to the cell surface, where it functions as a chloride channel and helps maintain the right balance of fluid in the airways.

  • Dirocaftor (PTI-808)

    Dirocaftor is a type of CFTR modulator called a potentiator. Potentiators help facilitate the opening of the chloride channel on the cell surface to allow chloride and sodium (salt) to move in and out of the cell. 

  • Riociguat

    Riociguat is a novel therapy that stimulates sGC, an enzyme in the cardiopulmonary system and the receptor for nitric oxide (NO). Preclinical data has shown evidence that riociguat can result in improved function of the CFTR protein as a chloride channel, moving salt and fluids in and out of cells.

     

  • VX-152 + tezacaftor + ivacaftor

    This program is testing VX-152 in combination with tezacaftor and ivacaftor. VX-152 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.

  • VX-440 + tezacaftor + ivacaftor

    This program is testing VX-440 in combination with tezacaftor and ivacaftor. VX-440 and tezacaftor (VX-661) are new CFTR correctors. Correctors are drugs designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. Once CFTR protein reaches the cell surface, ivacaftor helps facilitate the opening of the chloride channel to allow chloride and sodium (salt) to move in and out of the cell.

  • SION-638

    SION-638 is a corrector, a type of CFTR modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. It aims to stabilize defective CFTR by targeting a specific location on the protein called nucleotide binding domain 1, or NBD1. Once the stabilized CFTR protein reaches the cell surface, it forms a channel to allow chloride and sodium (salt) to move in and out of the cell. This new, unique approach could eventually offer an alternative to currently approved modulators.

  • SION-109

    SION-109 is a corrector, a type of CFTR modulator designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface. It aims to stabilize defective CFTR by targeting a specific location on the protein called intracellular loop 4, or ICL4. Once the stabilized CFTR protein reaches the cell surface, it forms a channel to allow chloride and sodium (salt) to move in an out of the cell. This approach could eventually offer an alternative to currently approved modulators to correct this aspect of the CFTR protein.

  • Sionna Therapeutics

    This program is developing potential CFTR modulators, a type of drug designed to fix the defective CFTR protein. These new modulators could benefit people with the most common CF-causing mutation, F508del, and may eventually offer an alternative to currently approved modulators.

  • Southern Research

    This program is working to identify potential therapies for nonsense mutations in the cystic fibrosis-causing gene. 

  • Icagen

    This program aimed to identify potential therapies for people with CF who have nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Overriding these premature stop signals would allow full-length, functional CFTR protein to be made.

  • Reata Pharmaceuticals

    This program aimed to expand the number of therapies designed to fix the defective CFTR protein in people who have the F508del mutation. 

Mucociliary Clearance

  • Dornase Alfa (Pulmozyme®)

    Dornase alfa (Pulmozyme®) is an inhaled medication that thins and loosens mucus in the airways of people with CF.

  • Hypertonic Saline

    Hypertonic saline as an inhaled therapy is believed to increase hydration of airway surface liquid in people with CF, thereby improving mucus clearance.

  • Inhaled Mannitol (Bronchitol®)

    Bronchitol® is an inhaled dry powder form of mannitol, a naturally occurring osmotic agent, which works by drawing water into the airways. This helps to moisten and thin the sticky mucus found in the lungs of people with CF, making it easier to cough it out.

  • BI 1265162

    BI 1265162 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • IONIS-ENaC-2.5

    IONIS-ENaC-2.5Rx is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.

  • OligoG

    OligoG is a dry powder drug that has been shown to decrease the thickness of mucus in the lungs and may help individuals with cystic fibrosis clear mucus easier. OligoG may also help improve the effectiveness of some antibiotics. It is administered using a dry powder inhaler and also developed as a liquid for use with a nebulizer.

  • QBW276

    QBW276 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • SPX-101

    SPX-101 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • VX-371 formerly (P-1037)

    VX-371 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • AZD5634

    AZD5634 is a compound designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance. 

  • GDC-6988 (formerly ETD002)

    GDC-6988 (formerly ETD002) is a compound designed to increase the activity of TMEM16A, a chloride channel found on the surfaces of airway cells. Similar to the CFTR channel, the TMEM16A channel helps regulate the amount of salt and fluids on the airway surface. Increasing the activity of TMEM16A may help increase the amount of fluid within the airways to improve mucus clearance.

  • ARO-ENaC

    ARO-ENaC is a drug intended to thin airway surface liquid. It is designed to block the function of the sodium (Na+) channel found in the lungs. These channels move sodium and water away from the airway surface. Blocking these channels may help maintain fluid within the airways to improve mucus clearance.

  • Denufosol

    This  alternative chloride channel activator has been removed from the pipeline due to lack of efficacy.

Anti-Inflammatory

  • High dose ibuprofen for CF

    Ibuprofen is an oral, non-steroidal, anti-inflammatory medication.

  • Acebilustat (CTX-4430)

    Acebilustat (CTX-4430) is an oral anti-inflammatory drug that reduces production of leukotriene B4 (LTB4), a molecule that leads to inflammation and is known to be elevated in people with CF. Reduction of inflammation helps prevent permanent tissue damage in the lungs.

  • Alpha 1 Anti-Trypsin

    Alpha 1 Anti-trypsin is an aerosolized protease inhibitor with anti-inflammatory properties, derived from human plasma.

  • Docosahexaenoic Acid (DHA)

    Docosahexaenoic acid (DHA) is an omega-3 fatty acid with anti-inflammatory properties.

  • Lenabasum (JBT-101)

    Lenabasum (JBT-101) is an oral drug that is aimed at promoting the resolution of inflammation. It is thought to increase production of anti-inflammatory molecules while reducing production of molecules that increase inflammation. Reduction of inflammation helps prevent permanent tissue damage in the lungs.

  • BI-1291583

    BI 1291583 is a drug designed to reduce inflammation in the lungs of people with bronchiectasis, the progressive lung disease that occurs in cystic fibrosis and other conditions. In CF, bronchiectasis is driven by a vicious cycle of mucus buildup, infection, and inflammation that damages the lungs over time.

    BI 1291583 works by inhibiting cathepsin C (CatC), an enzyme that exists naturally in the body and plays a key role in inflammation. When CatC is inhibited, or prevented from doing its job, the inflammation pathway is disrupted. This could allow BI 1291583 to reduce inflammation in the lungs of people with CF, improve symptoms, and help prevent permanent lung damage.

  • Brensocatib

    Brensocatib is an oral drug designed to block the function of enzymes, such as neutrophil elastase, that play an essential role in inflammation. When the lungs are infected with bacteria, white blood cells release these enzymes, including neutrophil elastase, to fight the infection. In CF, these enzymes can result in excessive inflammation that causes damage to the lungs. Brensocatib may decrease this damage by inhibiting neutrophil elastase and decreasing inflammation in the lungs.

  • LAU-7b

    LAU-7b is an oral compound. It is a form of the retinoid fenretinide. Retinoids are a group of compounds related to vitamin A. LAU-7b was studied for its potential to reduce the inflammatory response in the lungs of people with CF.

  • GS-5745

    GS-5745 is an antibody that may help reduce inflammation in the lungs, leading to improved lung function in people with CF.

  • KB001-A

    KB001-A is a humanized monoclonal Fab fragment that targets a Pseudomonas aeruginosa virulence factor (Type III secretion system). 

  • Sildenafil

    Sildenafil is a phosphodiesterase inhibitor. Researchers at National Jewish Health in Denver examined whether sildenafil can lower markers of airway inflammation in people with CF.

  • CB-280

    CB-280 is an oral drug designed to increase the amount of arginine in the lungs. Arginine is a molecule that occurs naturally in the body, and is important for the lungs to produce nitric oxide, a gas that helps the lungs fight infection.

    Sputum from people with CF has been shown to contain lower amounts of arginine and nitric oxide than normal. Lower nitric oxide levels are associated with worsened lung function and increased infection. Increasing arginine levels may increase the production of nitric oxide, which would strengthen the immune response, reduce inflammation and improve lung function.

  • Lonodelestat (formerly POL6014)

    Lonodelestat is a compound designed to block the function of neutrophil elastase. Neutrophil elastase is an enzyme that plays an essential role in inflammation. When the lungs are infected with bacteria, white blood cells release enzymes, including neutrophil elastase, to fight the infection. In CF, these enzymes can result in excessive inflammation that causes damage to the lungs. Lonodelestat was studied for its potential to block the function of neutrophil elastase, minimize tissue destruction, and decrease inflammation within the CF airway. 

  • PUR 118

    This compound is an inhaled dry powder formulation of a cationic airway liquid modulator.  It has anti-inflammatory properties and increases mucociliary clearance.

Anti-Infective

  • Amikacin Liposome Inhalation Suspension (Arikayce®)

    Amikacin liposome inhalation suspension (Arikayce®) is an antibiotic to treat chronic lung infections caused by nontuberculous mycobacteria (NTM). 

  • Azithromycin

    Azithromycin is an oral antibiotic with anti-inflammatory properties.

  • Aztreonam (Cayston®)

    Inhaled aztreonam (Cayston®) is an antibiotic that has been approved for people with CF who are chronically infected with Pseudomonas aeruginosa.

  • Inhaled Tobramycin

    TOBI® and  BETHKIS® are aerosolized forms of the antibiotic tobramycin, which is used to treat people with CF who are chronically infected with Pseudomonas aeruginosa. 

  • Tobramycin Inhaled Powder (TOBI® Podhaler™)

    The TOBI® Podhaler™ is the powder form of the inhaled antibiotic tobramycin and is used to treat people with CF who have Pseudomonas aeruginosa. It is taken with an inhaler, making it a convenient alternative to TOBI®, which must be taken using a nebulizer.

  • Inhaled Levofloxacin (Quinsair™)

    Inhaled levofloxacin (Quinsair™) is a formulation of the antibiotic levofloxacin for the management of chronic lung infections caused by Pseudomonas aeruginosa and other bacteria.

  • Inhaled Colistin (ColiFin®)

    Inhaled colistin is an antibiotic being studied to potentially treat chronic Pseudomonas aeruginosa infections in people with cystic fibrosis. Although it is approved in Europe, inhaled colistin is not approved for use in the U.S. Currently, the only approved inhaled antibiotics in the U.S. are tobramycin and aztreonam. However, many people with CF find that over time these therapies become less effective at treating their infections. Additionally, because there are few oral antibiotics that treat Pseudomonas, people with CF often must go on IV antibiotics when they have pulmonary exacerbations, a sudden worsening of respiratory symptoms caused by lung infections. IV antibiotics are problematic because they can cause hearing loss and kidney problems. Inhaled colistin could provide an additional option to help people with CF who are not responding to current treatments.

  • Vancomycin Inhalation Powder (AeroVanc™)

    Vancomycin inhalation powder (AeroVanc™) is an inhaled dry powder version of the antibiotic vancomycin for the treatment of methicillin-resistant Staphylococcus aureus (MRSA) airway infection in people with CF.

  • Intravenous Gallium

    Gallium is a molecule, nearly identical to iron, that disrupts iron-dependent biological processes and has been shown to kill antibiotic-resistant strains of Pseudomonas aeruginosa in laboratory research. Gallium has already been approved by the FDA for intravenous use in people. It is currently being studied for its effectiveness in treating infections in people with CF.

  • AP-PA02

    This program is studying AP-PA02, a type of phage therapy designed to fight Pseudomonas aeruginosa infections in people with CF. Bacteriophages are specialized viruses that kill very specific bacterial strains. They are found in the environment and are the predators of bacteria in nature. “Phage therapy” refers to the use of these bacteriophages to treat an infection in a person.

    AP-PA02 is a mix of multiple different types of phages that is also known as an inhaled cocktail. In lab tests AP-PA02 was able to kill more than 80 percent of Pseudomonas strains from people with CF.

  • AR-501 (Panaecin™)

    AR-501 (Panaecin™) is an inhaled formulation of gallium. Gallium is a molecule, nearly identical to iron, that disrupts iron-dependent biological processes and has been shown to kill antibiotic-resistant strains of Pseudomonas aeruginosa in laboratory research.

    Gallium can also be administered intravenously. Intravenous (IV) gallium has already been approved by the FDA for use in people and is now being studied for its safety and effectiveness in controlling infections in people with CF.

  • BX004-A

    This program is studying BX004-A, a type of phage therapy intended to treat Pseudomonas aeruginosa infections in the lung. Bacteriophages are specialized viruses that kill very specific bacterial strains. They are found in the environment and are the predators of bacteria in nature. “Phage therapy” refers to the use of these bacteriophages to treat an infection in a person.

    In lab studies, BX004-A was shown to be active against antibiotic-resistant strains of P. aeruginosa and demonstrated the ability to penetrate biofilm, a naturally forming structure that makes bacteria more resistant to antibiotics.

  • Inhaled Nitric Oxide (LungFit™ GO)

    LungFit™ GO is a portable inhaled nitric oxide treatment that is being tested for treatment of nontuberculous mycobacteria (NTM), a difficult-to-treat bacteria that infects the lungs of people with CF. Nitric oxide is a gas molecule produced by the body that plays a key role in the immune system. Nitric oxide has been shown to kill bacteria and break down their biofilms -- protective layers formed by bacteria that make them more difficult to eliminate. Researchers believe that increasing levels of nitric oxide in the body could help eliminate bacteria and increase lung function in people with CF. 

  • Opelconazole

    This program is developing an inhaled drug (opelconazole) to prevent Aspergillus fungal infections in lung transplant recipients. 

    There are many challenges associated with current treatment options for people infected with Aspergillus. They may be hard to tolerate, which may lead many patients to discontinue use before they have completed the treatment’s course. Some current treatments may also interact with other common transplant medicines, resulting in potentially serious side effects.

    This program will study whether inhaled opelconazole will be well tolerated and less likely to have drug-drug interactions. As an inhaled treatment option, it has the potential for being administered at home, instead of at a transplant or CF care center.

  • SPI-1005

    SPI-1005 is an oral form of the drug ebselen. Ebselen is a molecule designed to mimic the function of a key enzyme found in the inner ear, glutathione peroxidase (GPx). Regular use of aminoglycoside antibiotics can cause damaging side effects in the inner ear. By mimicking the function of GPx, SPI-1005 may protect the inner ear from damage.

  • Inhaled Molgramostim

    Inhaled molgramostim is a form of granulocyte macrophage-colony stimulating factor (GM-CSF). GM-CSF is a protein that occurs naturally in the human immune system and plays an important role in activating the immune system to kill bacteria and other infections. Because inhaled mogramostim helps to stimulate the part of the immune system that fights Nontuberculous mycobacteria (NTM), it may be able to help the body clear NTM infection.

  • Inhaled Nitric Oxide (Thiolanox®)

    Nitric oxide is a gas molecule produced by the body that plays a key role in the immune system. Nitric oxide has been shown to kill bacteria and break down their biofilms -- protective layers formed by bacteria that make them more difficult to eliminate. Researchers believe that increasing levels of nitric oxide in the body could help eliminate bacteria and increase lung function in people with CF. 

  • ACG-701 (formerly ARV-1801)

    ACG-701 (formerly ARV-1801) is a version of sodium fusidate. It is a compound that is used to treat methicillin-resistant Staphylococcus aureus, a cause of some CF lung infections, and may also decrease inflammation and mucus production. Sodium fusidate has been used in Europe, but it has never been approved in the U.S. This potential treatment comes in oral tablet form.

  • CMTX-101

    CMTX-101 is an anti-infective therapy that aims to disrupt bacterial biofilms, one of the primary causes of antibiotic resistance. Biofilms are protective structures that shield bacteria from both the immune system and antibiotics. 

    CMTX-101 specifically targets biofilms, potentially leaving bacteria more susceptible to antibiotics and the body’s own immune response. This approach could also help reduce the inflammation that results from chronic infections in CF. In lab tests, CMTX-101 was able to disrupt biofilms across many species of bacteria, including key CF pathogens such as Pseudomonas aeruginosa, Burkholderia cepacia, and nontuberculous mycobacteria. 

  • Lefamulin (Xenleta™)

    Lefamulin (Xenleta™) is a compound that interferes with bacteria’s ability to make protein, which is required for bacteria to grow. It is available in both oral and intravenous (IV) formulations. Lefamulin is FDA-approved for the treatment of adults with community-acquired bacterial pneumonia (CABP). It is currently being studied for its safety and pharmacokinetics in fighting infections in people with CF. 

  • RSP-1502

    This program is developing and testing a new inhaled antibiotic therapy for cystic fibrosis. This therapy is a combination of two existing approved drugs: the antibiotic tobramycin and another compound, calcium EDTA. When bacteria infect the lungs of people with CF, they can form structures called biofilms. Biofilms make bacteria more resistant to antibiotics. The compound calcium EDTA may help break down biofilms in the lungs, allowing antibiotics to fight bacteria more effectively.

  • ALX-009

    ALX-009 is a combination of two substances found naturally in the body, hypothiocyanite (OSCN-) and lactoferrin. Both of these molecules are part of the body’s natural immune system, but they are deficient in the airway surface liquid of people with CF. By adding both OSCN- and lactoferrin to the airways, ALX-009 may increase the ability of people with CF to fight bacteria without harming their body’s own cells. ALX-009 is delivered via inhalation.

  • SNSP113

    SNSP113 is an inhaled glycopolymer, a type of molecule being developed to treat infection and inflammation in the lungs. When bacteria infect the lungs of people with CF, it can form structures called biofilms. Biofilms make bacteria more resistant to antibiotics. SNSP113 helps break up biofilms in the lungs, allowing antibiotics to fight bacteria more effectively. SNSP113 may also make mucus thinner and less sticky, reducing inflammation and improving airway clearance. 

  • Inhaled Murepavadin

    This program is studying an inhaled version of murepavadin, an antibiotic that targets multi-drug resistant Pseudomonas aeruginosa infections in people with cystic fibrosis. An inhaled version could make it easier for someone with a Pseudomonas infection to take the drug from home. In addition, the drug, which targets the outer membrane of bacteria, has the potential to be a once-a-day treatment.

  • Kinnear Pharmaceuticals

    This program is studying CSA-131, a synthetic compound that mimics natural compounds in the body that help fight bacterial and fungal infections. CSA-131 aims to inhibit the growth of bacteria and fungi, break down their biofilms -- protective layers that bacteria and fungi form that make them more difficult to kill with anti-infectives -- and ultimately kill these organisms.

  • TB Alliance

    This program will identify and test potential treatments for infections caused by Mycobacterium abscessus (M. abscessus) and Mycobacterium avium complex (MAC), two types of nontuberculous mycobacteria (NTM). 

  • Matinas BioPharma

    This program studied MAT2501, an oral version of the drug amikacin. Amikacin is an antibiotic that targets nontuberculous mycobacteria (NTM) infections in people with cystic fibrosis. MAT2501 was studied for the potential to reduce the risk of known amikacin side effects such as hearing loss and kidney problems.

  • Microbion Biosciences

    This program studied pravibismane, an inhaled antibiotic. In the lungs of people with CF, bacteria may form protective layers -- known as biofilms -- that make them more difficult to kill. Pravibismane was studied for the potential to help break down biofilms and kill drug-resistant bacteria, such as multi-drug resistant Pseudomonas aeruginosa.

Nutritional-GI

  • AquADEKs

    AquaADEKs® is an oral antioxidant vitamin formulation specifically for people with CF.

  • Pancrelipase Enzyme Products

    These are digestive enzymes that are prepared using pancreases from pigs.

  • RELiZORB®

    RELiZORB® is a digestive enzyme cartridge that connects to enteral tube feeding systems. It is designed to mimic normal pancreatic function by helping to break down the fats in enteral tube feeding formula. By breaking down these fats before the formula is ingested, RELiZORB® may allow for delivery of increased absorbable calories and simplify the use of enzymes in overnight feedings. 

  • Liprotamase

    Liprotamase (Sollpura) is a pancreatic enzyme replacement that is not prepared from animal sources.

  • Burlulipase

    Burlulipase is a liquid solution developed to treat pancreatic insufficiency caused by CF. It is a bacterial lipase that has been shown to be more resistant against inactivation by gastric acid, giving the drug the potential to be more effective than existing pancreatin products derived from pigs. 

  • Glutathione

    Glutathione is an antioxidant that is important to the normal functioning of the intestine and lungs. Glutathione levels have been shown to be lower in people with CF. Oral glutathione may improve growth and decrease gut inflammation in children with CF.

  • Adrulipase (formerly MS1819-SD)

    Adrulipase (formerly MS1819-SD) is a non-porcine, meaning not pig-derived, enzyme for individuals with CF who have exocrine pancreatic insufficiency. It is a man-made version of a lipase enzyme taken from the yeast Yarrowia lipolytica. This drug does not contain any animal products.

  • ANG003

    This program is studying ANG003 (formerly SNSP003), a non-porcine (not pig-derived) enzyme replacement therapy for individuals with CF who have exocrine pancreatic insufficiency. It is a man-made version of a lipase enzyme taken from bacteria, and it does not contain any animal products.

    ANG003 may improve dosing convenience by reducing pill burden compared to current porcine-based pancreatic enzyme replacement therapies (PERT). ANG003 is also being developed in a formulation for pediatric and adult patients who are unable to swallow capsules.

To advance drug development and a search for a cure, the Cystic Fibrosis Foundation (CFF) has contracts with several companies to help fund the development of potential treatments and/or cures for cystic fibrosis. Pursuant to these contracts, CFF may receive milestone based payments, equity interests, royalties on the net sales of therapies, and/or other forms of consideration. Resulting revenue received by CFF is used in support of our mission. See “How Drugs Get on the Pipeline.” for more.

Sign up for clinical trial alerts

Get email updates about clinical trials that matter to you.

Explore clinical trials

Be a part of the movement transforming the future of cystic fibrosis treatment.

Learn More