Cystic fibrosis

Genetic Testing Market to Reach $28.24 Billion by 2030 - Market Size, Share, Forecasts, & Trends Analysis Report by Meticulous Research®

Retrieved on: 
Monday, March 20, 2023
Glaucoma, DNA, Ventricular septal defect, Prevalence, Impact, Viral Research and Diagnostic Laboratories, Parent, Hospital, Pharmacogenomics, COVID-19, Cancer, Down syndrome, Genetic testing, Diagnosis, Genomics, Hemoglobinopathy, Disorder, Government, PerkinElmer, Fragile X syndrome, MUFG Americas Holdings Corporation, Thermo Fisher Scientific, Patient, Laboratory, Risk, Developed country, Preimplantation genetic diagnosis, Investment, Labcorp, Consumables, Cystic fibrosis, Carrier, Hospital Clinic Manuel Quintela, Association for Molecular Pathology v. Myriad Genetics, Inc., Rare, Illumina, Inc., Geography, Pharmaceutical industry, Dietary supplement, Chromosome, Prenatal development

Genetic testing is done to look at changes in the DNA, sometimes called variants or mutations, that can lead to chronic diseases.

Key Points: 
  • Genetic testing is done to look at changes in the DNA, sometimes called variants or mutations, that can lead to chronic diseases.
  • Genetic testing is performed for different reasons, including pre-symptomatic and predictive testing, pharmacogenetics, diagnostic testing, carrier testing, prenatal testing, preimplantation testing, and newborn screening.
  • Genetic testing has applications in different areas of healthcare and has various advantages, such as determining the likelihood of parents passing the genetic mutation to their offspring.
  • Quick Buy – Genetic Testing Market Research Report: https://www.meticulousresearch.com/Checkout/33822956
    Based on end user, in 2023, the hospitals & clinics segment is expected to account for the largest share of this market.

Genetic Testing Market to Reach $28.24 Billion by 2030 - Market Size, Share, Forecasts, & Trends Analysis Report by Meticulous Research®

Retrieved on: 
Monday, March 20, 2023
Glaucoma, DNA, Ventricular septal defect, Prevalence, Impact, Viral Research and Diagnostic Laboratories, Parent, Hospital, Pharmacogenomics, COVID-19, Cancer, Down syndrome, Genetic testing, Diagnosis, Genomics, Hemoglobinopathy, Disorder, Government, PerkinElmer, Fragile X syndrome, MUFG Americas Holdings Corporation, Thermo Fisher Scientific, Patient, Laboratory, Risk, Developed country, Preimplantation genetic diagnosis, Investment, Labcorp, Consumables, Cystic fibrosis, Carrier, Hospital Clinic Manuel Quintela, Association for Molecular Pathology v. Myriad Genetics, Inc., Rare, Illumina, Inc., Geography, Pharmaceutical industry, Dietary supplement, Chromosome, Prenatal development

Genetic testing is done to look at changes in the DNA, sometimes called variants or mutations, that can lead to chronic diseases.

Key Points: 
  • Genetic testing is done to look at changes in the DNA, sometimes called variants or mutations, that can lead to chronic diseases.
  • Genetic testing is performed for different reasons, including pre-symptomatic and predictive testing, pharmacogenetics, diagnostic testing, carrier testing, prenatal testing, preimplantation testing, and newborn screening.
  • Genetic testing has applications in different areas of healthcare and has various advantages, such as determining the likelihood of parents passing the genetic mutation to their offspring.
  • Quick Buy – Genetic Testing Market Research Report: https://www.meticulousresearch.com/Checkout/33822956
    Based on end user, in 2023, the hospitals & clinics segment is expected to account for the largest share of this market.

Public Advisory - Two lots of cystic fibrosis drug Cayston recalled due to the potential of cracked glass vials

Retrieved on: 
Saturday, March 18, 2023
DIN, Sodium chloride, Sepsis, Abscess, Health status of Asian Americans, Urinary tract infection, Pharmacy, Patient, Risk, Cystic fibrosis, Health, Health Canada, Glass, Pneumonia, Pharmaceutical industry, Medical device, Aztreonam

Health Canada is advising the public that Gilead Sciences Incorporated recalled two lots (032168 and 033357) of the cystic fibrosis drug Cayston (aztreonam) (DIN 02329840) on March 1, 2023 , due to the potential of cracked glass vials in those lots, which may introduce glass particles into the medication.

Key Points: 
  • Health Canada is advising the public that Gilead Sciences Incorporated recalled two lots (032168 and 033357) of the cystic fibrosis drug Cayston (aztreonam) (DIN 02329840) on March 1, 2023 , due to the potential of cracked glass vials in those lots, which may introduce glass particles into the medication.
  • A cracked glass vial could cause potential injury or lead to potential contamination or a reduced dose.
  • Cayston is a prescription antibacterial medicine used for the management of chronic infections in cystic fibrosis patients.
  • The manufacturer has not received any customer complaints in relation to cracked glass vials in the recalled lots, or any other Cayston lots distributed in Canada.

Prime Therapeutics and Magellan Rx to present seven managed care pharmacy research studies at AMCP conference

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Thursday, March 16, 2023
Ranibizumab, DME, Research, Cancer, AMCP, Machine learning, PBM, Therapy, Depression, Cholesterol, Diabetic retinopathy, Commercial, Pharmacy, Prime, Maintenance, Pharmacotherapy, Medicare, Patient, SP, Academy of Managed Care Pharmacy, AMD, Cystic fibrosis, MID, Blue, Medicine, Single (music), Cryptocurrency, Health care, Pharmaceutical industry, Birth control, Health insurance, Bevacizumab

EAGAN, Minn., March 16, 2023 /PRNewswire/ -- Leading pharmacy benefit manager (PBM) Prime Therapeutics LLC (Prime) and Magellan Rx Management, (Magellan Rx), a Prime Therapeutics company, will present seven research studies at the Academy of Managed Care Pharmacy (AMCP) Annual Meeting, March 21-24 in San Antonio, Texas.

Key Points: 
  • EAGAN, Minn., March 16, 2023 /PRNewswire/ -- Leading pharmacy benefit manager (PBM) Prime Therapeutics LLC (Prime) and Magellan Rx Management , (Magellan Rx), a Prime Therapeutics company, will present seven research studies at the Academy of Managed Care Pharmacy (AMCP) Annual Meeting , March 21-24 in San Antonio, Texas.
  • Prime and Magellan Rx used integrated medical and pharmacy claims data to evaluate real-world drug utilization and managed care pharmacy programs.
  • "The teams conducting this research at Prime and Magellan Rx include some of the brightest minds in managed care pharmacy focused on whole-person care to not only improve health outcomes but also save health care costs," said Pat Gleason, PharmD, assistant vice president for health outcomes at Prime.
  • "These real-world studies demonstrate that with combined expertise, Prime and Magellan Rx are transforming the pharmacy industry, driving savings and bridging the distance between medical and pharmacy management to close gaps in care and help keep members healthier."

4D Molecular Therapeutics Reports Full Year 2022 Financial Results and Operational Highlights

Retrieved on: 
Wednesday, March 15, 2023
DME, EMA, Roche, Conference, Cystic fibrosis, Annual general meeting, VG, D, Macular degeneration, Diabetic retinopathy, Risk, PRISM, Fabry, Cohort, GMP, ARVO, Security (finance), CMC, Fabry disease, Patient, Cash, Research, FDA, AMD, CST, Safety, Clinical trial, IND, Vaccine, Pharmaceutical industry, 3D printing, Cryptocurrency, Ophthalmology

Our five clinical-stage product candidates address unmet medical needs in seven patient populations, including several large and high incidence rate disease markets.

Key Points: 
  • Our five clinical-stage product candidates address unmet medical needs in seven patient populations, including several large and high incidence rate disease markets.
  • R&D Expenses: Research and development expenses were $80.3 million for 2022, as compared to $61.4 million for 2021.
  • G&A Expenses: General and administrative expenses were $32.9 million for 2022, as compared to $28.0 million for 2021.
  • Net Loss: Net loss was $107.5 million for 2022, as compared to $71.3 million for 2021.

Inspire Biotherapeutics Launches with Pre-seed Investment to Target Monogenic and Acquired Lung Diseases with Proprietary AAVenger Gene Therapy Platform

Retrieved on: 
Wednesday, March 15, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, AAV, Regenerative medicine, Pulmonary fibrosis, Inspiration, Ontario Institute for Cancer Research, Monogenic, Mediterranean Sea, Clinical trial, Cystic fibrosis, Lung, Medical device, Medical imaging

Inspire Biotherapeutics Inc. (“Inspire”), an emerging biotech company with a mission to create life-saving gene therapies for monogenic and acquired diseases of the lung, is poised to move its novel lung tropic AAV gene therapy into clinical validation.

Key Points: 
  • Inspire Biotherapeutics Inc. (“Inspire”), an emerging biotech company with a mission to create life-saving gene therapies for monogenic and acquired diseases of the lung, is poised to move its novel lung tropic AAV gene therapy into clinical validation.
  • The AAVenger platform has demonstrated transduction and long-term expression in lung tissue without impact on other tissue types in disease-relevant animal models.
  • The platform can be applied to treat cystic fibrosis, interstitial lung disease, pulmonary fibrosis, and other monogenic lung diseases.
  • “Our lead indication is currently fatal in neonates – we are compelled to get this across the finish line!

Pediatric Medical Devices Market Report 2023: Sector to Reach $51.9 Billion by 2030 at a 7.8% CAGR - ResearchAndMarkets.com

Retrieved on: 
Wednesday, March 15, 2023
Medical Devices, Health, Quality of life, Epilepsy, Segment, Federal Food, Drug, and Cosmetic Act, IVD, Hospital, Prevalence, USD, Chapter Five, Patient, Conditional sentence, Kid, Personal protective equipment, COVID-19, Cardiology, FDA, Asthma, Cystic fibrosis, Diabetes, Child care, Medical device, Birth control, Nursing

The "Pediatric Medical Devices Market Size, Share & Trends Analysis Report By Product (Cardiology Devices, In Vitro Diagnostic (IVD) Devices, Diagnostic Imaging Devices), By End-user, By Region, And Segment Forecasts, 2023 - 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Pediatric Medical Devices Market Size, Share & Trends Analysis Report By Product (Cardiology Devices, In Vitro Diagnostic (IVD) Devices, Diagnostic Imaging Devices), By End-user, By Region, And Segment Forecasts, 2023 - 2030" report has been added to ResearchAndMarkets.com's offering.
  • The global pediatric medical devices market size is expected to reach USD 51.9 billion by 2030, expanding at a CAGR of 7.8% over the forecast period.
  • In the U.S., the commercialization and development of pediatric medical devices lag significantly behind the medical devices for adults.
  • Chapter 3 Pediatric Medical Devices Market: Variables, Trends, & Scope
    Chapter 4 Pediatric Medical Devices Market: Product Segment Analysis
    4.1 Pediatric Medical Devices Market: Market Share Analysis, 2022 & 2030
    Chapter 5 Pediatric Medical Devices Market: End-User Segment Analysis
    5.1 Pediatric Medical Devices Market: Market Share Analysis, 2022 & 2030

Nanite Inc. Receives Up to $2M Investment from CF Foundation to Explore a Novel Method to Deliver Genetic Therapies to the Lung

Retrieved on: 
Wednesday, March 15, 2023
Health, Technology, Genetics, Clinical Trials, Artificial Intelligence, Pharmaceutical, Biotechnology, PNP, Virus, Pancreas, Cystic fibrosis, Nanoparticle, Cystic Fibrosis Foundation, Nuclear power plant, Cell, Tissue, Patient, Gene, RNA, CF, Inc., Artificial intelligence, AI, Entrepreneurship, Lung, Pharmaceutical industry, Molecular machine

Nanite Inc. announced today an investment from the Cystic Fibrosis Foundation of up to $2 million to develop gene delivery technologies for the lung.

Key Points: 
  • Nanite Inc. announced today an investment from the Cystic Fibrosis Foundation of up to $2 million to develop gene delivery technologies for the lung.
  • One of the most significant challenges in developing new therapies for patients with cystic fibrosis is effective drug delivery.
  • Polymer nanoparticles (PNPs) are an innovative delivery modality and can offer several advantages over other means of genetic therapy delivery into cells.
  • “We’re thrilled to announce this investment from the Cystic Fibrosis Foundation, which we will use to explore a new way to deliver genetic therapies into the lungs of CF patients,” said Sean Kevlahan, CEO and Co-Founder, Nanite.

ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Trial of Novel Disease-Modifying Genetic Medicine, RCT1100 for the Treatment of Primary Ciliary Dyskinesia

Retrieved on: 
Wednesday, March 15, 2023
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Conference, DSM-IV codes, Disease, Messenger, Quality of life, Therapy, SORT, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, LNP, Trial of the century, Cell, PARI, CFTR, International Conference on Emerging Infectious Diseases, Human, MBA, Toxicology, ATS, Respiratory failure, PCD, Safety, Clinical trial, Primary ciliary dyskinesia, American Thoracic Society, Cystic fibrosis, Investigational New Drug, Food, IND, Bronchiectasis, Recode, Pharmacology, Vaccine, DNAI1

Patients with PCD have a high burden of morbidity with chronic respiratory infections and bronchiectasis, and often develop respiratory failure.

Key Points: 
  • Patients with PCD have a high burden of morbidity with chronic respiratory infections and bronchiectasis, and often develop respiratory failure.
  • RCT1100 is designed to be a disease-modifying mRNA-based therapeutic for PCD caused by pathogenic mutations in DNAI1, a gene that encodes a protein essential for ciliary movement.
  • The intent is for the mRNA delivery to lead to DNAI1 protein production in target cells and thereby rescue ciliary function.
  • The study is being conducted in New Zealand and ReCode anticipates enrolling approximately 32 healthy adults who will receive a single dose of either placebo or RCT1100.

Aridis Meets Primary and Secondary Endpoints in Phase 2a Study of AR-501 in Cystic Fibrosis Patients

Retrieved on: 
Monday, March 13, 2023
Cystic fibrosis, CFTR, Cystic Fibrosis Foundation, Bacteria, Șaeș, Gallium, Pharmacokinetics, Associate, Pneumonia, Respiratory tract, European Medicines Agency, Doctor of Philosophy, EMA, PK, Johns Hopkins University, CF, MD, Patient, Pseudomonas aeruginosa, Food, Lung, Safety, Nature, FDA, Infection, LOS, FEV1, US Foods, Antibiotics, Pharmaceutical industry

LOS GATOS, Calif., March 13, 2023 (GLOBE NEWSWIRE) -- Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS) today announced preliminary top-line results from the randomized, double blinded, placebo-controlled Phase 2a study of AR-501, which evaluated the safety and pharmacokinetics of three ascending doses of AR-501 administered as an inhaled aerosol in cystic fibrosis (CF) patients with confirmed Pseudomonas aeruginosa bacterial and other potential infections. The study was conducted with funding support from the Cystic Fibrosis Foundation. AR-501 is being developed as a once-per-week inhaled dosing regimen that is self-administered using a commercially available nebulizer device.

Key Points: 
  • The study was conducted with funding support from the Cystic Fibrosis Foundation.
  • AR-501 is being developed as a once-per-week inhaled dosing regimen that is self-administered using a commercially available nebulizer device.
  • Three weekly inhaled doses of AR-501 at 6.4mg, 20mg, and 40mg dose levels were well tolerated in CF patients.
  • “Having met the primary and secondary endpoints of this study, we thank the Cystic Fibrosis Foundation for the tremendous support they’ve provided to help us complete this study” said Aridis’ CEO Vu Truong, PhD.