Cystic fibrosis

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

Retrieved on: 
Tuesday, June 21, 2022
Biotechnology, General Health, Health, Doctor of Philosophy, Solution, University of California, San Francisco, Hope, Science, Cystic fibrosis transmembrane conductance regulator, Technology, Biology, Path, Cystic fibrosis, Cystic Fibrosis Foundation, Research, Valencia CF Foundation, CFTR, Drug discovery, University, National, Mutation, Conditional sentence, Conference, Therapy, Pharmaceutical industry, Antibiotics, Foundation

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.

Global RNA Therapeutics Market Trends, Growth Drivers, and Growth Opportunities 2021-2022 & 2027 - ResearchAndMarkets.com

Retrieved on: 
Friday, June 17, 2022
Genetics, Pharmaceutical, Health, Oligonucleotide, DNA, RNA, Therapy, COVID-19, CDMO, Cystic fibrosis, Research, Spinal muscular atrophy, MicroRNA, Technology transfer, Vaccine, Fine chemical

The "Global RNA Therapeutics Growth Opportunities" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global RNA Therapeutics Growth Opportunities" report has been added to ResearchAndMarkets.com's offering.
  • This research service provides an overview of the global RNA therapeutics industry from 2021 to 2027, including emerging trends, growth drivers, and growth opportunities.
  • The ribonucleic acid (RNA) therapeutics market is expected to witness remarkable clinical progress as therapies target "undruggable" pathways.
  • Technological advantages, coupled with the growing need to build clinically superior portfolios in RNA therapeutics for a competitive edge, are anticipated to drive strategic partnerships among stakeholders in the RNA therapeutics industry.

NasoNeb is now a Division of Monaghan Medical Corporation

Retrieved on: 
Thursday, June 16, 2022
Solution, MMC, OPEP, Cystic fibrosis, Pharmacy, Asthma, COPD, Rhinitis, Research, Hospital, NDS, Growth, Physician, General manager, Quality of life, Patient, Aerosol burn, Polyp (medicine), GLOBE, Administration, Sale, Pharmaceutical industry

PLATTSBURGH, N.Y., June 16, 2022 (GLOBE NEWSWIRE) -- NasoNeb, Inc. is proud to announce their integration with Monaghan Medical Corporation (Monaghan Medical) in Plattsburgh, New York.

Key Points: 
  • PLATTSBURGH, N.Y., June 16, 2022 (GLOBE NEWSWIRE) -- NasoNeb, Inc. is proud to announce their integration with Monaghan Medical Corporation (Monaghan Medical) in Plattsburgh, New York.
  • Monaghan Medical is a leader in the development, manufacture, and distribution of medical devices that improve quality of life of patients with lower respiratory diseases, such as asthma, Cystic Fibrosis, and COPD.
  • NasoNeb, Inc. can now look forward to operational and shipping efficiencies that will contribute to the growth of the NasoNeb intranasal drug delivery system and nasal care solution product line.
  • The NasoNeb product line was acquired in 2018 from MedInvent, Inc. by NasoNeb, Inc., a Trudell Medical Group member.

Global $25 Billion CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2028 - ResearchAndMarkets.com

Retrieved on: 
Thursday, June 16, 2022
Health, Pharmaceutical, Prevalence, Association, Country, Patent, US, Cystic fibrosis, Cystic Fibrosis Foundation, Survival, Ivacaftor, Precision medicine, Company, Research, Growth, Disease, CFTR, Clinical trial, Price analysis, Vertex, Cystic Fibrosis Trust, Patient, Modulation, Organization, Face, Generic drug, Pharmaceutical industry, Elexacaftor/tezacaftor/ivacaftor

The "Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global CFTR Modulators Market Opportunity, Dosage, Price & Clinical Trials Insight 2028" report has been added to ResearchAndMarkets.com's offering.
  • The report provides comprehensive insight on clinical and non-clinical factors that are driving the global CFTR Modulators market and its impact on the global pharmaceutical market landscape.
  • This drug class has grown considerably from 2012, with up to four agents available in the global market.
  • Global CFTR Modulators Market Opportunity: > USD 25 Billion
    Global CFTR Modulators Market Insights: Current, Regional, and Future Outlook 2028
    CFTR Modulators Availability By Dosage, Patent, and Price Analysis
    CFTR Modulators Clinical Trials Insight By Country, Company, and Phase: > 30 Drugs

Aceragen announces dosing of first patient with ACG-701 in clinical trial for treatment of melioidosis

Retrieved on: 
Wednesday, June 15, 2022
United States biological defense program, Department, Infection, Burkholderia pseudomallei, Mortality, Therapy, Weapon, Government, PEP, DTRA, Trilogy International Partners, Unitary state, Rheumatology, Chemical substance, Disease, Melioidosis, United States Department of Defense, Clinical trial, MD, Immune system, Program, Farber disease, WMD, QIDP, History, Patient, Post-exposure prophylaxis, Cystic fibrosis, GLOBE, Organic acid, Pharmaceutical industry

Melioidosis is an infection caused by the pathogen Burkholderia pseudomallei, which can result in a serious and life-threatening disease.

Key Points: 
  • Melioidosis is an infection caused by the pathogen Burkholderia pseudomallei, which can result in a serious and life-threatening disease.
  • Treatment failure rates for these infections can exceed 40% with current standard of care, resulting in persistently high morbidity and mortality.
  • Melioidosis is a neglected infection with limited development programs focusing on reducing serious outcomes, said Carl Kraus, MD, Chief Medical Officer of Aceragen and the principal investigator of the program.
  • I am pleased that we will be able to address both concerns with the launch of this clinical trial.

Global Orphan Drugs Market Report to 2030 - Players Include Amgen, Bristol-Myers Squibb and Pfizer - ResearchAndMarkets.com

Retrieved on: 
Tuesday, June 14, 2022
Health, Pharmaceutical, Cystic fibrosis, Lists of diseases, Gaucher's disease, Multiple myeloma, Degenerative disease, AML, Pancreatic cancer, HAE, Hereditary angioedema, Ovarian cancer, DMD, Fabry disease, Growth, Hypoparathyroidism, Birth, Acute myeloid leukemia, Infection, Duchenne muscular dystrophy, Prevalence, Alzheimer's disease, Renal cell carcinoma, Hunter syndrome, Non-Hodgkin lymphoma, Haemophilia, Huntington's disease, CAGR, Pharmaceutical industry, Duchenne

The global orphan drugs market was valued at $1,40,000.0 million in 2020 and is projected to reach $4,35,686.3 million by 2030 registering a CAGR of 11.8% from 2021 to 2030.

Key Points: 
  • The global orphan drugs market was valued at $1,40,000.0 million in 2020 and is projected to reach $4,35,686.3 million by 2030 registering a CAGR of 11.8% from 2021 to 2030.
  • The pharmaceutical agents administered to treat, diagnose, and prevent life-threatening diseases (rare diseases) are known as orphan drugs.
  • In addition, favorable government policies, and availability of market exclusivity for orphan drugs developers further supplement the global orphan drugs market growth.
  • The global orphan drugs market is segmented on the basis of disease type, and region to provide a detailed assessment of the market.

Santhera Reports 2021 Annual Results

Retrieved on: 
Friday, June 10, 2022
MAA, Company, Greater, Workforce, US FDA, Prednisone, Commercial, SEAL, Review, Annual, COVID-19, Bone fracture, LR, Association, CMO, Marketing, CEST, CEO, Treasury, BST, Mass meeting, Duchenne muscular dystrophy, NDA, EGM, Annual report, Contract, Proxy, Highbridge Capital Management, DMD, Vamorolone, Patient, Congenital muscular dystrophy, LEROS, AGM, Pratteln, USD, LHON, Exercise, Cystic fibrosis, CHF, US, Program, LLC, Comment, CF, Therapy, FDA, Growth, Clinical trial, Safety, Tutoring, Cryptocurrency, Medical device, Pharmaceutical industry, Airline, Animal, Security (finance), Duchenne, Idebenone, EU

Based on the agreement with the French authorities, Santhera has supplied Raxone free of charge from August 2021 following its removal from the list of reimbursed drugs.

Key Points: 
  • Based on the agreement with the French authorities, Santhera has supplied Raxone free of charge from August 2021 following its removal from the list of reimbursed drugs.
  • In March 2021, Santhera announced promising clinical trial data from a Phase 1b trial of lonodelestat in cystic fibrosis (CF).
  • Additional details on the pipeline products and operational progress can be found in the Annual Report 2021.
  • CEO Dario Eklund, CFO Andrew Smith and CMO Dr. Shabir Hasham will discuss the 2021 financial results and comment on ongoing corporate developments.

Global Nucleic Acid Based Gene Therapy Market to 2031 - Featuring BioMedica, Transgene and Copernicus Therapeutics Among Others - ResearchAndMarkets.com

Retrieved on: 
Thursday, June 9, 2022
Health, Genetics, Aptamer, Cystic fibrosis, Thalassemia, Economics, Lists of diseases, Anti-transglutaminase antibodies, Research, Plasma protein binding, FEE, Anemia, HCV, Food, Food and Drug Administration Safety and Innovation Act, Government, Elijah Cummings Lower Drug Costs Now Act, Recombinant, NIH, Hepatitis B virus, Therapy, Foundation for Economic Education, FDA, Growth, Hospital, Diabetes, HBV, HIV, Investor, Cytomegalovirus, Acid, CMV, Haemophilia, Protein, Vaccine, Pharmaceutical industry

The nucleic acid-based gene therapy market consists of sales of nucleic acid-based gene therapy products and services.

Key Points: 
  • The nucleic acid-based gene therapy market consists of sales of nucleic acid-based gene therapy products and services.
  • The main technologies in nucleic acid-based gene therapy are anti-sence and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, aptamers, and others.
  • Rising financial support by the government and the companies is projected to drive the demand for nucleic acid-based gene therapy.
  • This scenario is anticipated to restrain the market growth of the nucleic acid-based gene therapy market.

Verona Pharma Completes Enrollment in Phase 3 ENHANCE-1 Trial Evaluating Ensifentrine for Maintenance Treatment of COPD

Retrieved on: 
Thursday, June 9, 2022
Cystic fibrosis, Caregiver, COPD, Goal, Company, Research, Cystic fibrosis transmembrane conductance regulator, Breathe Me, Degenerative disease, Annual report, Lists of disasters, Government, Inflammation, Maintenance, Chronic obstructive pulmonary disease, GCP, FDA, Growth, Cough, Patient, Health, DPI, COVID-19, Compliance, Pandemic, SEC, Clinical trial, Virus, NDA, Consultant, RPL554, Asthma, Safety, Pharmaceutical industry

Ensifentrine has demonstrated statistically significant and clinically meaningful improvements in both lung function and symptoms, including breathlessness, in Verona Pharmas Phase 2 clinical studies in patients with moderate to severe COPD.

Key Points: 
  • Ensifentrine has demonstrated statistically significant and clinically meaningful improvements in both lung function and symptoms, including breathlessness, in Verona Pharmas Phase 2 clinical studies in patients with moderate to severe COPD.
  • Verona Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapies for the treatment of respiratory diseases with significant unmet medical needs.
  • The Company is evaluating nebulized ensifentrine in its Phase 3 clinical program ENHANCE (Ensifentrine as a Novel inHAled Nebulized COPD thErapy) for COPD maintenance treatment.
  • Two additional formulations of ensifentrine are in Phase 2 development for the treatment of COPD: dry powder inhaler (DPI) and pressurized metered-dose inhaler (pMDI).

 Vertex to Participate in the Goldman Sachs 43rd Annual Healthcare Conference on June 15

Retrieved on: 
Wednesday, June 8, 2022
Biotechnology, Pharmaceutical, Health, Cystic fibrosis, Biology, Conference, Research, Duchenne, LinkedIn, Beta thalassemia, Vertex Pharmaceuticals, News, CF, YouTube, Pain, Vertex, VRTX, Goldman, Sickle cell disease, Duchenne muscular dystrophy, Twitter, Instagram, Goldman Sachs, Pharmaceutical industry, Science, Facebook, Innovation district

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company will participate in a fireside chat at the Goldman Sachs 43rd Annual Global Healthcare Conference on Wednesday, June 15, 2022 at 1:20 p.m. PT.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the company will participate in a fireside chat at the Goldman Sachs 43rd Annual Global Healthcare Conference on Wednesday, June 15, 2022 at 1:20 p.m. PT.
  • A live webcast of management's remarks will be available through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page.
  • A replay of the conference webcast will be archived on the company's website.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.