Cystic fibrosis

VIVUS Provides Update on Pipeline and Program Milestones

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星期一, 一月 8, 2024
Patient, Quality of life, HIV/AIDS, Bloating, Diarrhea, Inflammatory bowel disease, ABPM, Chronic pain, Enzyme replacement therapy, Cystic fibrosis, Heart, Degenerative disease, Exocrine pancreatic insufficiency, Comorbidity, Steatorrhea, Obesity, HIV, Amylase, Orthostatic hypertension, Phentermine, Heart failure, Exercise, Pharmaceutical industry

CAMPBELL, Calif., Jan. 08, 2024 (GLOBE NEWSWIRE) -- VIVUS LLC, a biopharmaceutical company committed to the development and commercialization of innovative therapies that focus on advancing treatments for patients with serious unmet medical needs, today announced pipeline updates and program milestones.

Key Points: 
  • CAMPBELL, Calif., Jan. 08, 2024 (GLOBE NEWSWIRE) -- VIVUS LLC, a biopharmaceutical company committed to the development and commercialization of innovative therapies that focus on advancing treatments for patients with serious unmet medical needs, today announced pipeline updates and program milestones.
  • “VIVUS has been continuously dedicated to addressing the therapeutic needs of patients with serious medical conditions and life-limiting diseases, including exocrine pancreatic insufficiency, obesity and pulmonary arterial hypertension,” said John Amos, Chief Executive Officer at VIVUS LLC.
  • QSYMIA® is now the number one branded oral product in the US for obesity treatment.
  • Moreover, VIVUS is actively developing pipeline products focusing on bone marrow transplant preparation (VI-0609) and diabetes treatments (VI-0809 and VI-0810).”

Essential Pharma acquires European rights to Colobreathe®(colistimethate sodium) from Teva

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星期一, 一月 8, 2024
Acquisition, Cystic fibrosis, Pseudomonas aeruginosa, Mucus, Infection, Patient, Bacteria, Medicine, Psoriatic arthritis, Pharmaceutical industry

Zug, Switzerland and Egham, UK – 8 January 2024 – Essential Pharma, an international specialty pharma group focused on ensuring that patients have sustainable access to low volume, clinically well-established pharmaceutical products across key therapeutic areas, announces that it has completed the acquisition of European rights to Colobreathe® from Teva Laboratories UK Limited.

Key Points: 
  • Zug, Switzerland and Egham, UK – 8 January 2024 – Essential Pharma, an international specialty pharma group focused on ensuring that patients have sustainable access to low volume, clinically well-established pharmaceutical products across key therapeutic areas, announces that it has completed the acquisition of European rights to Colobreathe® from Teva Laboratories UK Limited.
  • Colobreathe® is indicated for the management of chronic pulmonary infections due to Pseudomonas aeruginosa (PsA) in patients with cystic fibrosis aged 6 years and older.
  • Under the terms of the acquisition, Essential Pharma has acquired the rights to Colobreathe® across European markets; the product is currently marketed across more than 20 countries in Europe, including the UK.
  • Emma Johnson, CEO of Essential Pharma, commented: “Our singular focus is on ensuring patients can maintain sustainable access to vital medicines.

Vertex Provides Pipeline and Business Updates in Advance of Upcoming Investor Meetings

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星期日, 一月 7, 2024
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, SCD, Kingdom, Blue Shield, Conference, T1D, Sickle cell disease, Gene editing, Blue Cross, Pain, Messenger, EMA, FDA, PDUFA, Cystic fibrosis, Insulin, Vertex Pharmaceuticals, Abdominoplasty, European Medicines Agency, Sciatica, SAD, PNP, Glycated hemoglobin, Patient, TDT, Safety, Diabetes, Acceleration, Hypoglycemia, CFTR, VRTX, CHMP, Pharmaceutical industry

Epidemiology update: Vertex revised its estimates for the number of patients living with cystic fibrosis from ~88,000 to ~92,000 in the U.S., Europe, Australia, and Canada.

Key Points: 
  • Epidemiology update: Vertex revised its estimates for the number of patients living with cystic fibrosis from ~88,000 to ~92,000 in the U.S., Europe, Australia, and Canada.
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of March 30, 2024, for CASGEVY in TDT.
  • VX-993: Vertex also anticipates initiating a Phase 2 study with an oral formulation of VX-993 for the treatment of PNP in 2024.
  • A live webcast of management's remarks will be available through the Vertex website, www.vrtx.com , in the "Investors" section under the "News and Events" page.

Clarametyx Biosciences Announces $33M Series A Financing to Advance Anti-Biofilm Biologics for Serious Bacterial Infections

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星期五, 一月 5, 2024
Infectious Diseases, Health, Pharmaceutical, Clinical Trials, Medical Supplies, Cystic Fibrosis Foundation, Lead, Hospital, ClinicalTrials.gov, Nationwide, Research, Cystic fibrosis, Pancreas, Infection, Genetically modified bacteria, Biofilm, Safety, Bacteria, DSM-IV codes, Pharmaceutical industry

(“Clarametyx”), a clinical stage company developing targeted, immune-enabling biologic therapies to counter persistent infections associated with biofilms, today announced the successful completion of a $33 million Series A round.

Key Points: 
  • (“Clarametyx”), a clinical stage company developing targeted, immune-enabling biologic therapies to counter persistent infections associated with biofilms, today announced the successful completion of a $33 million Series A round.
  • Clarametyx was launched in 2020 to advance a novel, immune-enabling antibody technology licensed from Nationwide Children’s Hospital.
  • The company is evaluating its anti-biofilm technology in both therapeutic and preventive settings to address the global challenge of persistent bacterial infection and antibiotic resistance.
  • “We graciously welcome our new investors and appreciate the continued commitment of our existing investors.

Sail Biomedicines Provides Update on Research with Cystic Fibrosis Foundation and Pioneering Medicines

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星期五, 一月 5, 2024
Software, Research, General Health, Pharmaceutical, Data Management, Technology, Genetics, Artificial Intelligence, Health Technology, Clinical Trials, Science, Biotechnology, Health, RNA, Cystic Fibrosis Foundation, Nonsense, Messenger, Cystic fibrosis, Pancreas, Flagship Pioneering, MBA, Biomedicine, Infection, DSM-IV codes, CFTR

Sail Biomedicines, Inc., a Flagship Pioneering company and leader in RNA-based programmable medicines, today provided an update on its research funded by the Cystic Fibrosis Foundation (CF Foundation), with preclinical data that suggest that Sail’s Endless RNA™ (eRNA™) may offer the potential to treat the 10%-15% of people with CF for whom existing treatments are not an option.

Key Points: 
  • Sail Biomedicines, Inc., a Flagship Pioneering company and leader in RNA-based programmable medicines, today provided an update on its research funded by the Cystic Fibrosis Foundation (CF Foundation), with preclinical data that suggest that Sail’s Endless RNA™ (eRNA™) may offer the potential to treat the 10%-15% of people with CF for whom existing treatments are not an option.
  • Sail’s programmable eRNA platform is designed to enable the in vivo expression of any protein, potentially targeting any tissue.
  • Cystic fibrosis is a progressive, genetic disease caused by mutations in the CFTR gene that affect the lungs, pancreas, and other organs.
  • “We are extremely hopeful about the impact this work could have for the CF community.”
    “The collaboration between Pioneering Medicines and the Cystic Fibrosis Foundation aims to accelerate the path to potential treatments and cures for all people living with cystic fibrosis,” said Paul Biondi, President, Pioneering Medicines and Executive Partner, Flagship Pioneering.

BiomX Receives Orphan Drug Designation from the U.S. Food and Drug Administration for BX004 for the Treatment of Chronic Pulmonary Infection Caused by Pseudomonas aeruginosa in Patients with Cystic Fibrosis

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星期四, 一月 4, 2024
FDA, Disease, Mortality, Cystic fibrosis, Orphan, Pseudomonas aeruginosa, Patient, NESS, Pharmaceutical industry

and NESS ZIONA, Israel, Jan. 04, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced that its phage cocktail, BX004, has been granted Orphan Drug Designation (“ODD”) by the United States Food and Drug Administration (“FDA”), for the treatment of chronic pulmonary infection caused by Pseudomonas aeruginosa (or P. aeruginosa) in patients with cystic fibrosis (“CF”).

Key Points: 
  • and NESS ZIONA, Israel, Jan. 04, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced that its phage cocktail, BX004, has been granted Orphan Drug Designation (“ODD”) by the United States Food and Drug Administration (“FDA”), for the treatment of chronic pulmonary infection caused by Pseudomonas aeruginosa (or P. aeruginosa) in patients with cystic fibrosis (“CF”).
  • As a reminder, in August 2023, the FDA granted BX004 Fast Track designation for the treatment of chronic pulmonary infections caused by P. aeruginosa bacterial strains in patients with CF.
  • “We are pleased to announce that the FDA has granted BX004 Orphan Drug Designation, which underscores the pressing need to develop new and innovative treatment options for this vulnerable patient population,” said Jonathan Solomon, Chief Executive Officer of BiomX.
  • “Persistent and deadly pulmonary infections from P. aeruginosaremain a major source of morbidity and mortality for CF patients, andBX004 has been designed to address this significant unmet need.

First Wave BioPharma Chairman and CEO Issues Letter to Shareholders Highlighting Recent Accomplishments and Outlook for 2024

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星期四, 一月 4, 2024
IBD, Food, Economics, Artificial intelligence, Letter, FDA, Cystic fibrosis, Gastrointestinal disease, Stanford University, Gastroparesis, Niclosamide, BOCA, Steatorrhea, Growth, Investment, First wave, Patient, Pharmaceutical industry

BOCA RATON, Fla., Jan. 04, 2024 (GLOBE NEWSWIRE) -- First Wave BioPharma, Inc., (NASDAQ: FWBI), (“First Wave BioPharma” or the “Company”), a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced that James Sapirstein, Chairman and CEO of First Wave BioPharma, has issued a Letter to Stockholders highlighting the Company’s recent accomplishments and plans for 2024.

Key Points: 
  • BOCA RATON, Fla., Jan. 04, 2024 (GLOBE NEWSWIRE) -- First Wave BioPharma, Inc., (NASDAQ: FWBI), (“First Wave BioPharma” or the “Company”), a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced that James Sapirstein, Chairman and CEO of First Wave BioPharma, has issued a Letter to Stockholders highlighting the Company’s recent accomplishments and plans for 2024.
  • The close of 2023 has been an exciting period for First Wave BioPharma, highlighted by the recently announced potential business combination with ImmunogenX.
  • The non-binding term sheet includes a low seven-figure payment to First Wave BioPharma along with economics related to future milestones and royalties.
  • 2024 has the potential to be a year of significant growth for First Wave BioPharma, and once again, I thank all of our stockholders for your continued support.

Cystic Fibrosis Rescued by Reprogramming the Cell’s Secretory Machinery

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星期三, 一月 3, 2024
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Protein, Cell, Drug design, Human, Publishing, Cystic fibrosis, Diabetes, Doctor of Philosophy, Therapy, Cancer, Life expectancy, Mucus, Mutation, Patent Cooperation Treaty, BioRxiv, DSM-IV codes, Patient, CFTR, Pharmaceutical industry

Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the publishing of a study that brings promise for a cure to cystic fibrosis (CF).

Key Points: 
  • Porosome Therapeutics, Inc. (Porosome Therapeutics) today announced the publishing of a study that brings promise for a cure to cystic fibrosis (CF).
  • “We look forward to bringing such new, novel, safe, and curative treatments to market for people living with cystic fibrosis, diabetes, and other secretory disorders.”
    On December 9, 2023, the preprint entitled “ Reprogramming the Cells Secretory Machinery: A Cystic Fibrosis Rescue ” was published on bioRxiv.
  • Results demonstrate that mutation in porosome-associated CFTR also affects other proteins within the porosome secretory machine, thereby reducing mucus secretion.
  • “Our company research has aided in the ability to identify the critical role CFTR plays within the porosome secretory machinery.

4DMT and Arbor Biotechnologies Establish Partnership to Co-Develop and Co-Commercialize Next-Generation Genetic Medicines for CNS Diseases

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星期三, 一月 3, 2024
Biology, Entrepreneurship, Partnership, ALS, Gene editing, Cardiology, A101, Drug development, R100, Protein engineering, FDA, CRISPR, Cystic fibrosis, Disease, Tissue, Brain, PAMS, Vivisection, AAV, Artificial intelligence, DSM-IV codes, Ophthalmology, CNS, Engineering, Machine learning, Genome, Central nervous system, Patient, Therapy, Vaccine

Arbor also has expertise in CNS biology, CNS drug development and CNS therapeutic development.

Key Points: 
  • Arbor also has expertise in CNS biology, CNS drug development and CNS therapeutic development.
  • 4DMT has utilized its platform to invent customized AAV vectors for CNS tissues, and these vectors will be deployed in the partnership.
  • “CNS disorders include some of the most devastating diseases, many of which have a genetic origin.
  • We look forward to combining our complementary technologies and capabilities to power a new generation of potential genetic medicines.

4DMT and Arbor Biotechnologies Establish Partnership to Co-Develop and Co-Commercialize Next-Generation Genetic Medicines for CNS Diseases

Retrieved on: 
星期三, 一月 3, 2024
Biology, Entrepreneurship, Partnership, ALS, Gene editing, Cardiology, A101, Drug development, R100, Protein engineering, FDA, CRISPR, Cystic fibrosis, Disease, Tissue, Brain, PAMS, Vivisection, AAV, Artificial intelligence, DSM-IV codes, Ophthalmology, CNS, Engineering, Machine learning, Genome, Central nervous system, Patient, Therapy, Vaccine

Arbor also has expertise in CNS biology, CNS drug development and CNS therapeutic development.

Key Points: 
  • Arbor also has expertise in CNS biology, CNS drug development and CNS therapeutic development.
  • 4DMT has utilized its platform to invent customized AAV vectors for CNS tissues, and these vectors will be deployed in the partnership.
  • “CNS disorders include some of the most devastating diseases, many of which have a genetic origin.
  • We look forward to combining our complementary technologies and capabilities to power a new generation of potential genetic medicines.