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Cystic Fibrosis Foundation
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Country
Country
U.S.A
Address
Address
4550 Montgomery Ave. Suite 1100 N Bethesda, MD 20814
Telephone
Telephone
301-951-4422

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The net proceeds will be used to support the development of curative therapies by using Prime Editing to correct CFTR mutations at the natural genetic locus, for the treatment of Cystic Fibrosis (CF).


Lead Product(s): Undisclosed

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Cell and Gene therapy

Recipient: Prime Medicine

Deal Size: $15.0 million Upfront Cash: Undisclosed

Deal Type: Funding January 25, 2024

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The funding will advance ARCT-032, a novel messenger RNA (mRNA) therapeutic candidate formulated with Arcturus’ LUNAR® delivery technology for the treatment of Cystic Fibrosis.


Lead Product(s): ARCT-032

Therapeutic Area: Genetic Disease Product Name: ARCT-032

Highest Development Status: Phase IProduct Type: Large molecule

Recipient: Arcturus Therapeutics

Deal Size: $25.0 million Upfront Cash: Undisclosed

Deal Type: Funding September 26, 2023

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If the therapy is successful, people with CF would be able to decrease the number of enzyme pills they must take to digest food properly from a handful of pills to only one per meal.


Lead Product(s): Enzyme-based Therapy

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: PreclinicalProduct Type: Large molecule

Recipient: Anagram Therapeutics

Deal Size: $15.5 million Upfront Cash: Undisclosed

Deal Type: Funding April 04, 2023

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AR-501 is a novel, small molecule and an inhalable form of gallium citrate, which acts as an iron analog to starve bacteria of iron. Gallium is believed to inhibit multiple iron-dependent synthetic and metabolic pathways required for pathogenicity.


Lead Product(s): Gallium Citrate

Therapeutic Area: Genetic Disease Product Name: AR-501

Highest Development Status: Phase I/ Phase IIProduct Type: Small molecule

Recipient: Aridis Pharmaceuticals

Deal Size: $12.5 million Upfront Cash: Undisclosed

Deal Type: Funding December 12, 2022

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BiomX has been given grant to support a clinical trial of BX004, a phage therapy to fight Pseudomonas aeruginosa infections in people with cystic fibrosis (CF). In lab experiments conducted by BiomX, BX004 killed antibiotic-resistant strains of P. aeruginosa.


Lead Product(s): BX004

Therapeutic Area: Infections and Infectious Diseases Product Name: BX004

Highest Development Status: Phase I/ Phase IIProduct Type: Large molecule

Recipient: BiomX

Deal Size: $5.0 million Upfront Cash: Undisclosed

Deal Type: Funding January 05, 2022

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The award provided by the Cystic Fibrosis Foundation will generate both in vitro and in vivo data about PLG0301 and PLG0206, which may potentially support an Investigational New Drug (IND) application with the FDA for its continued development.


Lead Product(s): PLG0301

Therapeutic Area: Infections and Infectious Diseases Product Name: PLG0301

Highest Development Status: PreclinicalProduct Type: Peptide

Recipient: Peptilogics

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Funding January 05, 2022

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The CF Foundation’s funding will support Phase 2 study of opelconazole. The objective of the study is to assess the safety and tolerability and patient’s ability to complete the course of treatment when used to prevent invasive pulmonary fungal infections in lung transplant.


Lead Product(s): Opelconazole

Therapeutic Area: Infections and Infectious Diseases Product Name: PC945

Highest Development Status: Phase IIProduct Type: Small molecule

Recipient: Pulmocide

Deal Size: $3.5 million Upfront Cash: Undisclosed

Deal Type: Funding December 06, 2021

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Pioneering Medicines and the Cystic Fibrosis Foundation will partner with multiple companies within the Flagship ecosystem, leveraging emerging technologies including Tessera Therapeutics’ Gene WritingTM technology, targeted delivery modalities, and RNA technologies.


Lead Product(s): Undisclosed

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: DiscoveryProduct Type: Undisclosed

Recipient: Flagship Pioneering

Deal Size: $110.0 million Upfront Cash: Undisclosed

Deal Type: Partnership November 03, 2021

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This new funding of up to $1.8 million will directly support IND-enabling toxicology studies of pravibismane and is in addition to $5.6 million previously awarded by the Foundation, for a combined total of up to $7.4 million.


Lead Product(s): Pravibismane

Therapeutic Area: Infections and Infectious Diseases Product Name: MBN-101

Highest Development Status: IND EnablingProduct Type: Small molecule

Recipient: Microbion

Deal Size: $7.4 million Upfront Cash: Undisclosed

Deal Type: Funding February 22, 2021

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The funding will support a clinical trial to determine whether high doses of inhaled nitric oxide are safe and effective when used in conjunction with current standard-of-care therapies, including amikacin and azithromycin.


Lead Product(s): Nitric Oxide,Amikacin,Azithromycin

Therapeutic Area: Infections and Infectious Diseases Product Name: Undisclosed

Highest Development Status: UndisclosedProduct Type: Small molecule

Recipient: Beyond Air

Deal Size: $2.1 million Upfront Cash: Undisclosed

Deal Type: Funding February 16, 2021

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