Cystic fibrosis transmembrane conductance regulator

Vertex to Present Data Highlighting Long-Term Benefits of TRIKAFTA® at the North American Cystic Fibrosis Conference

Retrieved on: 
Thursday, November 2, 2023
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Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), which can treat the underlying cause of cystic fibrosis (CF) in ~90% of people with the disease, will be presented at the 2023 North American Cystic Fibrosis Conference (NACFC) taking place November 2-4, 2023 in Phoenix, Arizona.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), which can treat the underlying cause of cystic fibrosis (CF) in ~90% of people with the disease, will be presented at the 2023 North American Cystic Fibrosis Conference (NACFC) taking place November 2-4, 2023 in Phoenix, Arizona.
  • Presentations will highlight results from two open-label extension studies demonstrating the long-term clinical benefits and safety profile of TRIKAFTA® among CF pediatric populations.
  • Vertex will present interim data on TRIKAFTA® from its longest pediatric follow-up study to date.
  • The data also showed a continued favorable safety profile, with no new safety findings (Poster #133).

Sionna Therapeutics Presents Preclinical Data at 2023 North American Cystic Fibrosis Conference Demonstrating Series 2 NBD1 Stabilizers Normalize ΔF508-CFTR Function

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Friday, November 3, 2023
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BOSTON, Nov. 3, 2023 /PRNewswire/ -- Sionna Therapeutics, a clinical-stage life sciences company dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF), today announced the presentation of preclinical data on highly potent Series 2 nucleotide binding domain-1 (NBD1) stabilizers, SION-719 and SION-451. These data show that SION-719 and SION-451 fully restore maturation, trafficking, and function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to wild-type levels in combination with complementary modulators. The data are being presented at the 2023 North American Cystic Fibrosis Conference (NACFC) in Phoenix, Arizona.

Key Points: 
  • The data are being presented at the 2023 North American Cystic Fibrosis Conference (NACFC) in Phoenix, Arizona.
  • The most common CFTR mutation in CF is ΔF508, which results in NBD1 destabilization that contributes to defective CFTR folding, trafficking, half-life, and function.
  • Sionna is advancing small molecule NBD1 stabilizers, a novel therapeutic class with the potential to fully correct ΔF508-CFTR.
  • Sionna is also developing complementary intracellular loop 4 (ICL4) and transmembrane domain 1 (TMD1)-directed modulators to use in combination with NBD1 stabilizers.

Moligo Technologies and Nationwide Children's Hospital Collaborate to Advance Cystic Fibrosis Research Using Ultrapure Long Single-Stranded DNA

Retrieved on: 
Tuesday, October 24, 2023
Sequence, Sale, DNA, AAV, Vaidyanathan, CFTR, Research, Reactive airway disease, Risk, Crownsville Hospital Center, Gene, Cystic fibrosis, Nationwide Children's Hospital, Patient, Hospital, CF, B cell, Cystic fibrosis transmembrane conductance regulator, Vaccine

STOCKHOLM, Oct. 24, 2023 /PRNewswire/ -- Moligo Technologies announced today that it has signed a collaboration agreement with Nationwide Children's Hospital to provide long, single-stranded DNA for its research into gene therapies for cystic fibrosis (CF) and other airway diseases. Moligo's patent-pending enzymatic DNA production technology delivers ultrapure, long ssDDNA at industrial sale - with the ability to introduce chemical modifications - making it potentially suited as a non-viral delivery method in cell and gene therapies.

Key Points: 
  • STOCKHOLM, Oct. 24, 2023 /PRNewswire/ -- Moligo Technologies announced today that it has signed a collaboration agreement with Nationwide Children's Hospital to provide long, single-stranded DNA for its research into gene therapies for cystic fibrosis (CF) and other airway diseases.
  • While significant advances have been made in gene therapy, using the approach for airway diseases is still in the early stages.
  • Moligo utilizes a proprietary, PCR-free, enzymatic technology to produce ultrapure DNA strands over 10,000 bases long, 100 percent sequence-verified, and at scale.
  • Sequence complexity is also a significant challenge, making encoding many complete and functional long therapeutic genes into ssDNA impossible.

CF Foundation Invests Up to $9 Million in Arcturus Therapeutics to Develop a Messenger RNA Therapy

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Tuesday, September 26, 2023
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Arcturus is developing an mRNA therapy that is designed to deliver the mRNA template to the lung cells to produce functional cystic fibrosis transmembrane conductance regulator (CFTR) protein .

Key Points: 
  • Arcturus is developing an mRNA therapy that is designed to deliver the mRNA template to the lung cells to produce functional cystic fibrosis transmembrane conductance regulator (CFTR) protein .
  • “Messenger RNA therapy offers a way to treat all people with CF, especially those who can’t tolerate or don’t respond to modulator treatment,” said JP Clancy, MD, senior vice president of clinical research at the Foundation.
  • The additional $9 million in funding combined with the $15.6 million already provided to Arcturus brings our total commitment to nearly $25 million.
  • The Foundation is funding research into mRNA therapies because of their potential to treat all people with CF.

Vertex Receives CHMP Positive Opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor for Children With Cystic Fibrosis Ages 2 Through 5

Retrieved on: 
Friday, September 15, 2023
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Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of children with cystic fibrosis (CF) ages 2 through 5 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
  • “KAFTRIO has demonstrated unprecedented clinical benefit for eligible people living with CF,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex.
  • “Treating the underlying cause of CF as early as possible in life has the potential to slow disease progression, which is why we are pleased the CHMP is supportive of expanding the indication for KAFTRIO to patients as young as 2 years.”
    In the European Union, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is already approved for the treatment of people with CF ages 6 years and older who have at least one copy of the F508del mutation in the CFTR gene.

CENTOGENE Reports First Half 2023 Financial Results

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Thursday, September 7, 2023
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and ROSTOCK, Germany and BERLIN, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced its unaudited financial results for the six months ended June 30, 2023.

Key Points: 
  • and ROSTOCK, Germany and BERLIN, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced its unaudited financial results for the six months ended June 30, 2023.
  • Cost of sales increased by €2,692 thousand, or 20.7%, to €15,728 thousand for the first half of 2023, from €13,036 thousand for the first half of 2022.
  • General administrative expenses decreased by €112 thousand, or 0.6%, to €17,172 thousand for the first half of 2023, from €17,284 thousand for the first half of 2022.
  • The decrease is mainly due to the increase in consumable expenses on approximately €1,601 thousand for the first half of 2023.

4DMT Presents Positive Interim Data from Aerosolized 4D-710 Phase 1/2 AEROW Clinical Trial in Patients with Cystic Fibrosis at the ECFS 46th Annual Meeting

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Wednesday, June 7, 2023
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ET with cystic fibrosis specialist Dr. Jennifer L. Taylor-Cousar

Key Points: 
  • ET with cystic fibrosis specialist Dr. Jennifer L. Taylor-Cousar
    EMERYVILLE, Calif., June 07, 2023 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT, or the Company), a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines, today announced positive interim clinical data from the 4D-710 Phase 1/2 AEROW clinical trial for the treatment of cystic fibrosis lung disease.
  • 4D-710 is comprised of our targeted and evolved vector, A101, and a codon-optimized CFTR∆R transgene and is designed for aerosol delivery to achieve CFTR expression within lung airway epithelial cells.
  • Interim data from the study will be presented in an oral presentation titled, “AAV mediated gene therapy for cystic fibrosis: Interim results from a Phase 1/2 clinical trial,” at the European Cystic Fibrosis Society (ECFS) 46th Annual Meeting held in Vienna, Austria on Thursday, June 8 at 5:00 p.m. CET (11:00 a.m.
  • “We believe that 4D-710 represents a potentially transformative new treatment for people with CF.

Vertex Presents Data Demonstrating Significant Benefits of Long-Term Treatment With CFTR Modulators at the European Cystic Fibrosis Conference

Retrieved on: 
Friday, June 9, 2023
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Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that 12 scientific abstracts on the company’s portfolio of cystic fibrosis (CF) medicines were presented at this year’s European Cystic Fibrosis Society's (ECFS) 46th European Cystic Fibrosis Conference held June 7-10, 2023, in Vienna, Austria.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that 12 scientific abstracts on the company’s portfolio of cystic fibrosis (CF) medicines were presented at this year’s European Cystic Fibrosis Society's (ECFS) 46th European Cystic Fibrosis Conference held June 7-10, 2023, in Vienna, Austria.
  • Together, the data presented show the long-term benefits of treatment with CFTR modulators as well as the importance of treating the underlying cause of CF as early in life as possible.
  • Key data presented at this year’s conference are highlighted below.
  • “The data on TRIKAFTA in particular demonstrate that this medicine improves lung function sustainably and in a real-world setting.

Vertex Announces U.S. FDA Approval for KALYDECO® (ivacaftor) to Treat Eligible Infants With CF Ages 1 Month and Older

Retrieved on: 
Wednesday, May 3, 2023
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KALYDECO® is already approved in the U.S. and EU for the treatment of CF in patients ages four months and older.

Key Points: 
  • KALYDECO® is already approved in the U.S. and EU for the treatment of CF in patients ages four months and older.
  • This cohort demonstrated a safety profile similar to that observed in older children and adults.
  • KALYDECO® was first approved in 2012 in the U.S. and is now available in more than 30 countries.
  • For more information on KALYDECO®, prescribing information, or patient assistance programs, visit Kalydeco.com or VertexGPS.com .

Vertex Announces U.S. FDA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 2 Through 5 With Certain Mutations

Retrieved on: 
Wednesday, April 26, 2023
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“Since its initial approval, TRIKAFTA has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.

Key Points: 
  • “Since its initial approval, TRIKAFTA has had a significant impact on the CF community, transforming the lives of thousands of people living with cystic fibrosis,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.
  • The data from this study were recently published in the American Journal of Respiratory and Critical Care Medicine .
  • Additionally, Vertex has submitted applications for the use of TRIKAFTA®/KAFTRIO® in children ages 2 through 5 years of age to other global regulatory authorities, including the European Medicines Agency (EMA) and the Medicines and Healthcare Products Regulatory Agency (MHRA).
  • For more information on TRIKAFTA®, patient assistance programs or to find additional eligibility details, visit TRIKAFTA.com , VertexGPS.com or vertextreatments.com .