Cystic fibrosis

Vast Therapeutics Expands Leadership Team with Appointment of Dr. Paul Bruinenberg as Chief Medical Officer

Retrieved on: 
Thursday, March 7, 2024
Rochester University, Bronchiectasis, Roche, Cystic fibrosis, MD, NIH, Medical director, Nephrotoxicity, Astellas Pharma, MBA, TB, ARUP Laboratories, University of Utah, University, Therapy, Pathology, TB Alliance, Associate, Patient, Duke University, Diagnosis, Tuberculosis, Pseudomonas aeruginosa, NTM, Publication, IND, Pharmaceutical industry

MORRISVILLE, N.C., March 7, 2024 /PRNewswire/ -- Vast Therapeutics today announced the addition of Paul Bruinenberg, MD, MBA as Chief Medical Officer. Dr. Bruinenberg brings over 30 years of experience as a clinical development executive in respiratory medicine and orphan diseases. This appointment and the recent addition of microbiologist and clinical diagnostics expert Chris Polage, MD, as Medical Director, significantly bolster the company's clinical development expertise in pulmonary infectious diseases and medical microbiology. 

Key Points: 
  • MORRISVILLE, N.C., March 7, 2024 /PRNewswire/ -- Vast Therapeutics today announced the addition of Paul Bruinenberg, MD, MBA as Chief Medical Officer.
  • "Paul's development experience will guide Vast Therapeutics as we successfully transition to a clinical-stage organization," said Nate Stasko, Chief Executive Officer at Vast Therapeutics.
  • "It is an amazing opportunity to join a company with an accepted IND for a novel treatment modality that shows promising efficacy against chronic infections caused by Pseudomonas aeruginosa and non-tuberculosis mycobacteria," commented Paul Bruinenberg, Chief Medical Officer at Vast Therapeutics.
  • Most recently, Dr. Bruinenberg served as the Senior Medical Officer of the TB Alliance leading clinical development against mycobacterial infections.

New York State Department of Health Selects HealthWell Foundation Subsidiary to Administer its Adult Cystic Fibrosis Assistance Program

Retrieved on: 
Monday, March 4, 2024
EST, Pharmacy, Communication, Medicare, Patient, Medicaid, Cystic fibrosis, Insurance, Partnership

GERMANTOWN, Md., March 4, 2024 /PRNewswire/ -- The HealthWell Foundation®, an independent non-profit charitable organization that provides financial assistance for underinsured Americans, is pleased to announce that its wholly-owned subsidiary, HWF Direct, LLC, has been engaged to administer New York State's Adult Cystic Fibrosis Assistance Program (ACFAP).

Key Points: 
  • GERMANTOWN, Md., March 4, 2024 /PRNewswire/ -- The HealthWell Foundation®, an independent non-profit charitable organization that provides financial assistance for underinsured Americans, is pleased to announce that its wholly-owned subsidiary, HWF Direct, LLC, has been engaged to administer New York State's Adult Cystic Fibrosis Assistance Program (ACFAP).
  • Assisting the Cystic Fibrosis community has been a longstanding commitment at the HealthWell Foundation and HWF Direct.
  • We are excited to strengthen our impact on the CF community through our partnership with the New York State Department of Health."
  • To apply for a grant through New York State's Adult Cystic Fibrosis Assistance Program, contact HWF Direct Monday – Friday, 9:00 a.m. – 5:00 p.m. EST at 1-855-226-2295.

THE BIG SWELL IS COMING: PAPA'S PILAR RUM ANNOUNCES NEWEST LEGACY EDITION IN HONOR OF SURFING LEGEND GREG NOLL

Retrieved on: 
Wednesday, February 28, 2024
Cystic fibrosis, Papa, Sherry, Legacy series, Life, Human, Ernest Hemingway, WWII, KEY, Heart, Taste, Noll, Film industry

KEY WEST, Fla., Feb. 28, 2024 /PRNewswire/ --Today, Papa's Pilar Rum, the worldly-sourced and Florida-finished rum brand inspired by legendary novelist Ernest "Papa" Hemingway, announces availability of the latest rum in its Legacy series, Legacy Edition 2023, crafted in honor of surfing legend and big wave pioneer Greg Noll.

Key Points: 
  • KEY WEST, Fla., Feb. 28, 2024 /PRNewswire/ --Today, Papa's Pilar Rum, the worldly-sourced and Florida-finished rum brand inspired by legendary novelist Ernest "Papa" Hemingway, announces availability of the latest rum in its Legacy series, Legacy Edition 2023, crafted in honor of surfing legend and big wave pioneer Greg Noll.
  • Papa's Pilar Rum introduces Legacy Edition 2023, crafted in honor of surfing legend and big wave pioneer Greg Noll.
  • "Much like Ernest Hemingway, Greg Noll was larger than life; he was a surfing trailblazer but also a committed craftsman.
  • "Working with the Papa's Pilar team and Hemingway family on this one-of-a-kind rum to celebrate my father has been an honor."

Championing Rare Voices on Rare Disease Day

Retrieved on: 
Monday, February 26, 2024
Rare disease, Education, Physician, Quality of life, Federation, Hope, Takeda, Rare Disease Day, Patient, Disease, Diagnosis, Cystic fibrosis, Partnership

Recently, 13 Canadian rare disease organizations, in partnership with Takeda Canada Inc. ("Takeda"), united to launch I Am Number 12 - a national campaign dedicated to sharing the unique stories and perspectives of rare disease Changemakers, and to show that rare diseases aren't really that rare.

Key Points: 
  • Recently, 13 Canadian rare disease organizations, in partnership with Takeda Canada Inc. ("Takeda"), united to launch I Am Number 12 - a national campaign dedicated to sharing the unique stories and perspectives of rare disease Changemakers, and to show that rare diseases aren't really that rare.
  • The initiative continues to amplify the voices of those in the community to encourage conversations on Rare Disease Day.
  • "Rare Disease Day is a symbol of hope for millions of Canadians impacted by rare diseases.
  • "Takeda is honoured to partner with rare disease organizations who work tirelessly to advocate for the one in twelve Canadians affected by a rare disease."

Royalty Pharma Reports Q4 and Full Year 2023 Results

Retrieved on: 
Thursday, February 15, 2024
MorphoSys, Pfizer, European Commission, Recurrence, Docetaxel, Roche, Cystic fibrosis, Share repurchase, FDA, Food, Schizophrenia, Growth, Sacituzumab govitecan, PTC, Vertex, Astellas Pharma, Risdiplam, Metastasis, Projection, KarXT, Bristol Myers Squibb, PTC Therapeutics, Endocrine gland, Lung cancer, Civil service commission, JAK, Therapy, Tourette syndrome, Webcast, Patient, Cytokinetics, Eastern Time Zone, Novartis, Ruxolitinib, NDA, Pharmaceutical industry

In October 2023, Royalty Pharma drew $350 million under its existing unsecured revolving credit facility, which was repaid during the fourth quarter of 2023.

Key Points: 
  • In October 2023, Royalty Pharma drew $350 million under its existing unsecured revolving credit facility, which was repaid during the fourth quarter of 2023.
  • Royalty Pharma began repurchasing its Class A ordinary shares in April 2023 under a $1.0 billion multi-year share repurchase program.
  • During the fourth quarter of 2023, Royalty Pharma announced new transactions of up to $1.6 billion.
  • Royalty Pharma will host a conference call and simultaneous webcast to discuss its fourth quarter and full year 2023 results today at 8:00 a.m., Eastern Time.

Intellia Therapeutics and ReCode Therapeutics Announce Strategic Collaboration to Develop Novel Gene Editing Therapies for Cystic Fibrosis

Retrieved on: 
Thursday, February 15, 2024
Therapy, LNP, Liver, MB, CRISPR, Gene editing, Messenger, Intellia Therapeutics, Recode, Cystic fibrosis, Mucus, NTLA, Lung, DSM-IV codes, Respiratory failure, Cell, CFTR, Patient, Medicine, Tissue, Research, DNA, Vaccine

and MENLO PARK, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).

Key Points: 
  • and MENLO PARK, Calif., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).
  • Intellia will be responsible for the design of the editing strategy and research-grade components for the investigational therapies.
  • “This collaboration provides further validation of ReCode's SORT LNP platform to deliver diverse gene editing modalities to specific cells and tissues.
  • By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients.”

Intellia Therapeutics and ReCode Therapeutics Announce Strategic Collaboration to Develop Novel Gene Editing Therapies for Cystic Fibrosis

Retrieved on: 
Thursday, February 15, 2024
Health, Genetics, Other Science, Research, Science, Biotechnology, Therapy, LNP, Liver, MB, CRISPR, Gene editing, Intellia Therapeutics, Messenger, Recode, Cystic fibrosis, Mucus, NTLA, DSM-IV codes, Respiratory failure, Cell, CFTR, Patient, Medicine, Tissue, DNA, Vaccine

Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).

Key Points: 
  • Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, and ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced a strategic collaboration to develop novel genomic medicines for the treatment of cystic fibrosis (CF).
  • Intellia will be responsible for the design of the editing strategy and research-grade components for the investigational therapies.
  • “This collaboration provides further validation of ReCode's SORT LNP platform to deliver diverse gene editing modalities to specific cells and tissues.
  • By combining our highly synergistic technologies and capabilities, we are excited about the potential to enable a faster path for next-generation gene editing therapeutics to CF patients.”

EQS-News: Investor interest returns – dividend yield remains high for 2024

Retrieved on: 
Wednesday, February 14, 2024
Acquisition, Annual general meeting, RSV, USD, Cystic fibrosis, Ionis, FDA, Pain, Patient, Policy, BB Biotech, Alnylam Pharmaceuticals, Depression, Pharmaceutical industry

Release as at February 14, 2024

Key Points: 
  • Release as at February 14, 2024
    After a difficult 2023 stock market year, above all due to higher interest rates, the capital markets are now once again looking at biotech companies with interest.
  • The prospect of falling interest rates has led to a rethink.
  • A dividend of this magnitude is exceptional not just in the Swiss equity market, but also in an international comparison.
  • Experts are anticipating similarly high approval figures for 2024 too.

Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor/Tezacaftor/Deutivacaftor, Next-In-Class Triple Combination Treatment for Cystic Fibrosis

Retrieved on: 
Monday, February 5, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, CFTR, Patient, Consultant, Safety, VRTX, Mutation, Vertex, Chairperson, Disease, Therapy, Cystic fibrosis, Medicine, University, Vertex Pharmaceuticals

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”) program, the most comprehensive Phase 3 pivotal program ever conducted by Vertex for the treatment of cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced positive results from its once-daily vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”) program, the most comprehensive Phase 3 pivotal program ever conducted by Vertex for the treatment of cystic fibrosis (CF), a progressive, multi-organ disease caused by dysfunction of the CFTR protein.
  • In both SKYLINE 102 and SKYLINE 103, the primary endpoint of absolute change from baseline in ppFEV1 through week 24 was met and showed that treatment with vanza triple was non-inferior to treatment with TRIKAFTA.
  • The results from other secondary endpoints were consistent with results of the primary and key secondary endpoints.
  • Treatment with the vanza triple was well tolerated in all three studies, and the safety was similar between the vanza triple and TRIKAFTA treatment groups in SKYLINE 102 and SKYLINE 103.

Enterprise Therapeutics Closes £26 million ($33.1 million) Series B Follow-on Financing

Retrieved on: 
Tuesday, January 30, 2024
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Urinary tract infection, Chronic obstructive pulmonary disease, CFTR, Mortality, FEV1, Respiratory disease, Therapy, Disease, Patient, Enterprise, Mucus, IP Group, Cystic fibrosis, Epithelial sodium channel, Asthma, Atomic mass

Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering from respiratory disease, today announced that it has closed a £26 million ($33.1 million USD) Series B follow-on financing round, led by Panakes Partners.

Key Points: 
  • Enterprise Therapeutics Ltd (Enterprise), a biopharmaceutical company dedicated to the discovery and development of novel therapies to improve the lives of patients suffering from respiratory disease, today announced that it has closed a £26 million ($33.1 million USD) Series B follow-on financing round, led by Panakes Partners.
  • Existing investors Versant Ventures, Novartis Venture Fund, Forbion, Epidarex Capital and IP Group also participated.
  • Alongside the financing, Dr Rob Woodman, Partner at Panakes, joins Enterprise’s Board of Directors.
  • Dr John Ford, CEO, Enterprise Therapeutics, said: “We have made tremendous progress to date in developing novel therapeutics for patients suffering from chronic respiratory diseases.