Cystic fibrosis transmembrane conductance regulator

Prime Medicine Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 9, 2023
LNP, GAA, FXN, CMC, AAV, Chemistry, R, CF, Quarter, Stem cell, Administration, Patient, Chronic granulomatous disease, Mitochondrion, Eye, HSC, CFTR, Research and development, Research, Central nervous system, Liver, Swelling, Gene expression, Investment, Cell, WD, Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, FRDA, CGD, Pharmaceutical industry, Vaccine, Video game, Cryptocurrency, IND

CAMBRIDGE, Mass., March 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2022.

Key Points: 
  • “In 2022, we made important progress in our efforts to build Prime Medicine and demonstrate the promise of Prime Editors as a potentially best-in-class genetic medicine approach,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Initiate investigational new drug (IND)-enabling studies of PM359 for the treatment of CGD in 2023.
  • Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.
  • Fourth Quarter and Full Year 2022 Financial Results:
    Research and Development (R&D) Expenses: R&D expenses were $29.1 million for the fourth quarter of 2022 and $86.7 million for the year ended December 31, 2022, as compared to $52.9 million for the fourth quarter of 2021 and $70.6 million for the year ended December 31, 2021.

Prime Medicine Announces Recent Progress and Highlights 2023 Strategic Priorities

Retrieved on: 
Monday, January 9, 2023
Disease, FRDA, Genome, Cystic fibrosis transmembrane conductance regulator, Swelling, CGD, Safety data sheet, Respiratory tract, GAA, Tissue, Cystic fibrosis, CD34, CMC, AAV, Heart, Failure rate, CFTR, Mitochondrion, Culture, DNA, LNP, Safety, Mutation, IPSC, Gene editing, Central nervous system, Hepatocyte, Patient, Degenerative disease, CF, Bronchiole, Neuron, PCSK9, Ataxia, Liver, Human, FXN, Inflammation, Chronic granulomatous disease, Eye, Investment, Prime editing, Vaccine, Pharmaceutical industry, Medical device, IND, WD, Frataxin

CAMBRIDGE, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today provided an update on recent advancements of its Prime Editing technology and progress across its initial pipeline of eighteen programs, and outlined its strategic priorities and expected milestones for 2023.

Key Points: 
  • Today, Prime Medicine announced new preclinical data demonstrating that Prime Editing-mediated removal of pathological repeats in vitro results in correction of hypermethylation at the FXN gene, restoring genetic function back to wild-type levels.
  • Off-target Safety Data: Prime Medicine is progressing a comprehensive suite of assays to evaluate the potential off-target activity of its Prime Editors.
  • Prime Medicine is using PCSK9 as a model system for developing its modular LNP delivery platform to the liver.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Nominate first development candidate for CGD in 1Q 2023.

CF Foundation Invests Up to $15M in ReCode Therapeutics for Development of mRNA Therapy

Retrieved on: 
Tuesday, January 10, 2023
Biotechnology, Health, Other Health, Pharmaceutical, Other Philanthropy, Philanthropy, Other Science, Research, General Health, Fund Raising, Genetics, Foundation, Science, Clinical Trials, Cell, Cystic fibrosis transmembrane conductance regulator, Valencia CF Foundation, Cystic fibrosis, COVID-19, RNA transfection, Recode, CFTR, MD, Mutation, Cystic Fibrosis Foundation, Vaccine, LNP, Conference, RNA, CF, Research, Pharmaceutical industry

The CF Foundation joins other institutional and strategic investors who participated in ReCode’s Series B financing, totaling $210 million.

Key Points: 
  • The CF Foundation joins other institutional and strategic investors who participated in ReCode’s Series B financing, totaling $210 million.
  • ReCode is developing an inhaled mRNA therapy, designed to provide a correct copy of the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA to lung cells to make a functional CFTR protein .
  • These LNPs may allow genetic therapies to enter lung cells more easily and importantly may allow safe redosing of the CFTR mRNA.
  • In lab tests, ReCode demonstrated their mRNA therapy can be delivered in an aerosol form to human lung cells.

Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA

Retrieved on: 
Monday, December 12, 2022
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, Cystic fibrosis transmembrane conductance regulator, IND, Disease, Exocrine gland, Lung, Partnership, Death, Liver, Pain, Cell, Multimedia, Clinical trial, Annual report, FASN, Beta thalassemia, HBE, Cystic fibrosis, Security (finance), GI, Food, Woman, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Vertex Pharmaceuticals, Pancreas, Medication, LNP, Water, VRTX, Therapy, Inhalation, Outline, Degenerative disease, Sickle cell disease, Patient, Research, Biology, Medicine, RNA transfection, Safety, Genotype, Pharmaceutical industry, Vaccine, Biotechnology, Science, Vertex, Innovation district, CFTR, Moderna

Vertex plans to initiate a single ascending dose clinical trial for VX-522 in people with CF in the coming weeks.

Key Points: 
  • Vertex plans to initiate a single ascending dose clinical trial for VX-522 in people with CF in the coming weeks.
  • (Graphic: Business Wire)
    VX-522 is delivered to the lung through inhalation of a CFTR mRNA encapsulated by a lipid nanoparticle.
  • Once delivered to the target lung cells, the mRNA is designed to produce functional copies of the CFTR protein.
  • This investigational messenger ribonucleic acid (mRNA) therapy aims to address the underlying cause of cystic fibrosis (CF).

Sionna Therapeutics Announces IND Clearance and Phase 1 Initiation for SION-638 in Cystic Fibrosis

Retrieved on: 
Wednesday, December 7, 2022
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BOSTON, Dec. 7, 2022 /PRNewswire/ -- Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced the clearance of its Investigational New Drug application (IND) from the U.S. Food and Drug Administration (FDA) for SION-638, a novel small molecule designed to target the first nucleotide-binding domain (NBD1) of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The first subject has been dosed in a Phase 1 study to evaluate the safety and pharmacokinetics of SION-638 in healthy volunteers.

Key Points: 
  • The first subject has been dosed in a Phase 1 study to evaluate the safety and pharmacokinetics of SION-638 in healthy volunteers.
  • "We have achieved an important milestone of advancing SION-638, a first-in-class NBD1 modulator, into a Phase 1 study.
  • The most common mutation in CFTR, F508, causes NBD1 to unfold at body temperature and severely impairs CFTR function.
  • Sionna Therapeutics is a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF) by normalizing the function of CFTR, the key protein associated with disease progression in CF.

Vertex to Present New Data on its Portfolio of Cystic Fibrosis Medicines at the 2022 North American Cystic Fibrosis Conference

Retrieved on: 
Thursday, November 3, 2022
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These data will also be discussed during the second plenary on November 4, 2022, at NACFC.

Key Points: 
  • These data will also be discussed during the second plenary on November 4, 2022, at NACFC.
  • These long-term data at 144 weeks demonstrate remarkable health effects in the longest study of TRIKAFTA.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Sionna Therapeutics to Present at the North American Cystic Fibrosis Conference

Retrieved on: 
Tuesday, October 4, 2022
Cystic fibrosis, Entrepreneurship, Pennsylvania Convention Center, CF, Research, Cystic fibrosis transmembrane conductance regulator, Cystic Fibrosis Foundation, Mutation, CFTR, HIV disease progression rates, Therapy, Conference, Patient, Senior, Pharmaceutical industry, Sionna

BOSTON, Oct. 4, 2022 /PRNewswire/ -- Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced that it will present preclinical data on novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator combinations at the Cystic Fibrosis Foundation's North American Cystic Fibrosis Conference (NACFC) 2022 to be held November 3-5 at the Pennsylvania Convention Center in Philadelphia. These combinations include Sionna's first-in-class molecules that target the first nucleotide-binding domain (NBD1) of CFTR, with the potential to enable full restoration of CFTR function in CF patients with the ΔF508 mutation. 

Key Points: 
  • BOSTON, Oct. 4, 2022 /PRNewswire/ -- Sionna Therapeutics, a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced that it will present preclinical data on novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator combinations at the Cystic Fibrosis Foundation's North American Cystic Fibrosis Conference (NACFC) 2022 to be held November 3-5 at the Pennsylvania Convention Center in Philadelphia.
  • These combinations include Sionna's first-in-class molecules that target the first nucleotide-binding domain (NBD1) of CFTR, with the potential to enable full restoration of CFTR function in CF patients with the F508 mutation.
  • Sionna Therapeutics is a life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF) by normalizing the function of CFTR, the key protein associated with disease progression in CF.
  • Sionna's pipeline has the potential to deliver best-in-class efficacy and reach previously unachievable levels of long-term benefit for people with CF.

Krystal Biotech Announces FDA Acceptance of KB407 IND Application for Cystic Fibrosis Clinical Trial

Retrieved on: 
Monday, August 1, 2022
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KB407 is a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs.

Key Points: 
  • KB407 is a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs.
  • On July 1, 2022, the Company submitted an IND application to initiate the Phase 1 clinical trial of KB407.
  • At the end of the 30-day review period, the Company received notification that the FDA accepted the IND allowing the Phase 1 clinical trial to begin.
  • The Phase 1 clinical trial will utilize nebulized administration to deliver KB407 in up to 20 adults with CF.

Krystal Biotech Receives FDA Acceptance of KB407 IND Application for Cystic Fibrosis Clinical Trial

Retrieved on: 
Monday, August 1, 2022
Private Securities Litigation Reform Act, Death, United, KRYS, Spirometry, Skin, GLOBE, CF, Drug, Epithelium, Degenerative disease, U.S. Securities and Exchange Commission, Â, Twitter, Cystic fibrosis, Pancreas, Safety, NASDAQ, Lung, Drug discovery, Clinical trial, CFTR, Review, Disease, FDA, Company, Cystic Fibrosis Foundation, LinkedIn, Patient, Mucus, Senior, Lists of diseases, Cystic fibrosis transmembrane conductance regulator, Urinary tract infection, IND, Pharmaceutical industry, Antibiotics, Medical device

KB407 is a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs.

Key Points: 
  • KB407 is a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs.
  • On July 1, 2022, the Company submitted an IND application to initiate the Phase 1 clinical trial of KB407.
  • At the end of the 30-day review period, the Company received notification that the FDA accepted the IND allowing the Phase 1 clinical trial to begin.
  • The Phase 1 clinical trial will utilize nebulized administration to deliver KB407 in up to 20 adults with CF.

Cystic Fibrosis Foundation Invests $6M in Carbon Biosciences to Advance New Gene Therapy Approach for Cystic Fibrosis

Retrieved on: 
Tuesday, June 21, 2022
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The Cystic Fibrosis Foundation today announced its investment of $6 million in Carbon Biosciences to support the companys preclinical research into an innovative gene therapy approach for cystic fibrosis.

Key Points: 
  • The Cystic Fibrosis Foundation today announced its investment of $6 million in Carbon Biosciences to support the companys preclinical research into an innovative gene therapy approach for cystic fibrosis.
  • Carbon Biosciences began its scientific work in May at the CF Foundation Therapeutics Lab in Lexington, Mass., marking the first time the Foundation is hosting scientists from a startup company.
  • As the Foundation expands its laboratory footprint later this summer, it will add space dedicated to incubating new companies focusing on CF research.
  • The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis.