Cystic fibrosis transmembrane conductance regulator

ReCode Therapeutics Raises Oversubscribed Series B Financing Round of $80 Million

Retrieved on: 
Thursday, October 21, 2021
Professional Services, Health, Genetics, Finance, Clinical Trials, Pharmaceutical, Biotechnology, Series, RNA transfection, HBE, Primary ciliary dyskinesia, Technology, LinkedIn, CFTR, Twitter, DNAI1, Epithelium, Patient, RNA, Recode, B cell, Cystic fibrosis transmembrane conductance regulator, Cystic fibrosis, Company, CEO, Tissue, Cell, PCD, LNP, Therapy, CF, Mouse, Gene editing, Liver, OUP, Pharmaceutical industry, Vaccine

ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the closing of an $80 million Series B financing round co-led by Pfizer Ventures and EcoR1 Capital.

Key Points: 
  • ReCode Therapeutics (the Company), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the closing of an $80 million Series B financing round co-led by Pfizer Ventures and EcoR1 Capital.
  • Existing investors who participated included OrbiMed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures, L.P., and Osage University Partners (OUP).
  • Oleg Nodelman, founder and portfolio manager of EcoR1 Capital also joined ReCodes Board of Directors in connection with the financing.
  • ReCode Therapeutics is an integrated genetic medicines company developing disease-modifying therapeutics using its powerful LNP delivery technology to target organs and tissues beyond the liver.

Verona Pharma to Present Phase 2 Data with pMDI Ensifentrine in COPD at CHEST 2021

Retrieved on: 
Wednesday, October 13, 2021
Breathe Me, Company, Inflammation, Cystic fibrosis transmembrane conductance regulator, Cough, Lists of disasters, COVID-19, DPI, Growth, Cystic fibrosis, Annual report, SEC, Virus, Chronic obstructive pulmonary disease, Degenerative disease, Maintenance, COPD, Pandemic, Asthma, RPL554, Consultant, Patient, Research, Pharmaceutical industry

Ensifentrine also activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which is beneficial in reducing mucous viscosity and improving mucociliary clearance.

Key Points: 
  • Ensifentrine also activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which is beneficial in reducing mucous viscosity and improving mucociliary clearance.
  • 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 Chronic Obstructive Pulmonary Disease (COPD).
  • 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).

4D Molecular Therapeutics Announces FDA Clearance of IND Application for 4D-710, an A101 Vector-based, Aerosol-delivered Gene Therapy for the Treatment of Cystic Fibrosis Lung Disease

Retrieved on: 
Wednesday, October 6, 2021
Food, Fabry disease, Securities Act of 1933, Mucus, Patient, Choroideremia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Lung transplantation, Cystic fibrosis, Bronchiectasis, Drug Quality and Security Act, Inflammation, Security (finance), Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, FDA, AMD, Population, Valencia CF Foundation, Marketing, GLOBE, Urinary tract infection, Terminology, Ophthalmology, Entrepreneurship, Disease, Trial of the century, Risk, COVID-19, Human, IND, Tissue, CFTR, Cell, Safety, Forward-looking statement, AAV, Cystic fibrosis transmembrane conductance regulator, Securities Exchange Act of 1934, Cardiology, Epithelium, Mutation, Lung, Pharmaceutical industry, Vaccine, Medical imaging

4D-710 is an aerosol-delivered gene therapy that has promise as a mutation agnostic treatment for patients with cystic fibrosis lung disease, said David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT.

Key Points: 
  • 4D-710 is an aerosol-delivered gene therapy that has promise as a mutation agnostic treatment for patients with cystic fibrosis lung disease, said David Kirn, M.D., Co-Founder and Chief Executive Officer of 4DMT.
  • 4D-710 is comprised of our targeted and evolved vector, A101, and a microCFTR transgene, said Robert Fishman, M.D., Chief Medical Officer of 4DMT.
  • 4D-710 has the potential to be a differentiated therapy for the treatment of cystic fibrosis lung disease due to its potentially corrective mechanism, expected ability to treat patients independent of CFTR mutation and resistance to AAV antibodies.
  • Cystic fibrosis is the most common life-shortening inherited disease in the United States and results from mutations in the CFTR gene.

Krystal Biotech Receives Approval from the Human Research Ethics Committee in Australia for Phase 1 Trial of KB407 for Cystic Fibrosis

Retrieved on: 
Wednesday, September 29, 2021
Clinical trial, DSMB, Cohort, Data monitoring committee, LCI, Cystic fibrosis, CF, NASDAQ, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, Patient, Adult, Degenerative disease, Krystal, OMICS Publishing Group, Suma, Marketing, FEV1, MBW, Death, COVID, Company, Disease, Trial of the century, OGTR, COVID-19, Spirometry, Research, CFTR, Pancreas, Safety, Office of the Gene Technology Regulator, KRYS, Cystic fibrosis transmembrane conductance regulator, Genotype, Epithelium, Lung, GLOBE, Pharmaceutical industry, Antibiotics

PITTSBURGH, Sept. 29, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, announced today that it has been granted approval by the Bellberry Human Research Ethics Committee (HREC) in Australia to conduct a Phase 1 clinical study of inhaled KB407 in patients with cystic fibrosis (CF). The Company previously received license to evaluate KB407 from Australia's Office of the Gene Technology Regulator (OGTR). KB407 is an engineered viral vector designed to deliver full-length human cystic fibrosis transmembrane conductance regulator (CFTR) to the airways of people with cystic fibrosis via nebulization. Study enrollment is expected to begin shortly following approval by the trial site’s institutional governance and biosafety committee.

Key Points: 
  • The Company previously received license to evaluate KB407 from Australia's Office of the Gene Technology Regulator (OGTR).
  • KB407 is an engineered viral vector designed to deliver full-length human cystic fibrosis transmembrane conductance regulator (CFTR) to the airways of people with cystic fibrosis via nebulization.
  • Study enrollment is expected to begin shortly following approval by the trial sites institutional governance and biosafety committee.
  • Cystic fibrosis is the most common fatal inherited disease in the United States, affecting more than 80,000 patients worldwide.

Research Efforts Fuel the Future of Global Obstructive Lung Disease Management Markets - ResearchAndMarkets.com

Retrieved on: 
Wednesday, September 22, 2021
Biotechnology, General Health, Medical Devices, Other Health, Health, Obstructive lung disease, Patent, Asthma, RNA, Therapy, Chronic obstructive pulmonary disease, Hospital, Patient, Strategy, Method, Respiratory disease, Growth, Research, Self-management, CFTR, COPD, Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, Medical device, Pharmaceutical industry

The "Global Obstructive Lung Disease Management Research Report" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Obstructive Lung Disease Management Research Report" report has been added to ResearchAndMarkets.com's offering.
  • The report provides a glimpse into the near future of respiratory disease treatment by highlighting newer therapeutics and advanced technologies that can address existing challenges in obstructive lung disease management.
  • Obstructive lung diseases such as chronic obstructive pulmonary disease (COPD), asthma, and cystic fibrosis are a huge burden on the global healthcare system.
  • What are the growth opportunities for companies developing new therapeutics and technologies for obstructive lung disease management?

Verona Pharma Presents Pharmacokinetic Data with Ensifentrine at the European Respiratory Society International Congress 2021

Retrieved on: 
Thursday, September 2, 2021
Company, DPI, Degenerative disease, Virus, Growth, Lists of disasters, Consultant, SEC, Maintenance, Pandemic, Research, Chronic obstructive pulmonary disease, COPD, Asthma, Cystic fibrosis transmembrane conductance regulator, Cough, Inflammation, Breathe Me, Patient, RPL554, Annual report, COVID-19, Cystic fibrosis, Pharmaceutical industry

Ensifentrine also activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which is beneficial in reducing mucous viscosity and improving mucociliary clearance.

Key Points: 
  • Ensifentrine also activates the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), which is beneficial in reducing mucous viscosity and improving mucociliary clearance.
  • Ensifentrines mechanism of action has the potential to alleviate respiratory symptoms such as breathlessness and cough and work against inflammation associated with COPD or inflammation triggered by viruses.
  • 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.

Vertex Announces Publication in The New England Journal of Medicine of Phase 3 Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People With Cystic Fibrosis

Retrieved on: 
Thursday, August 26, 2021
Health, Genetics, Clinical Trials, General Health, Pharmaceutical, Biotechnology, VRTX, CF, University, University of Alabama, CI, CFTR, Ivacaftor, The New England Journal of Medicine, Cystic fibrosis, Mutation, Manuscript, Safety, Cystic fibrosis transmembrane conductance regulator, Patient, Vertex Pharmaceuticals, NEJM, Pharmaceutical industry

This study is the third of three Phase 3 clinical trials in the TRIKAFTA program in the 12 years and older age group.

Key Points: 
  • This study is the third of three Phase 3 clinical trials in the TRIKAFTA program in the 12 years and older age group.
  • These results are especially notable given that all patients were treated with a CFTR modulator prior to initiating TRIKAFTA.
  • Following the run-in, 258 patients were randomized to receive TRIKAFTA or to remain on their prior regimen of ivacaftor or tezacaftor/ivacaftor for 8 weeks.
  • Baseline was measured at the end of the run-in period, prior to the start of the 8-week treatment period.

Translate Bio Announces Second Quarter 2021 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, August 5, 2021
Company, Cf, Infection, Clinical trial, Data, American Thoracic Society, U.S. Securities and Exchange Commission, Investment, Vaccine, Collaboration, Patent, Ministry of Education (Malaysia), PCD, Private Securities Litigation Reform Act, Technology, Risk, DNAI1, Earnout, Sanofi Pasteur, IND, Partnership, Time, Pulmonology, Translation, Therapy, Animal, Patient, COVID-19, Twitter, MRT, Food and Drug Administration, Sanofi, Cystic fibrosis transmembrane conductance regulator, ATS, RNA, Sanofi–Translate Bio COVID-19 vaccine, Research, RNA transfection, Cystic fibrosis, Liver, Safety, Conference, Acquisition, Security (finance), GLOBE, Pharmaceutical industry

LEXINGTON, Mass., Aug. 05, 2021 (GLOBE NEWSWIRE) -- Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat or prevent debilitating or life-threatening diseases, today announced financial results for the second quarter ended June 30, 2021 and reviewed recent corporate updates.

Key Points: 
  • This transaction is a validation of our mRNA platform and a testament to the talented Translate Bio team.
  • Translate Bio ended the second quarter of 2021 with $667.2 million in cash, cash equivalents and investments and 75,343,712 shares of common stock outstanding.
  • General and administrative expenses of $11.9 million during the second quarter of 2021, compared to $8.6 million for the same period in 2020.
  • Translate Bio is also pursuing the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi Pasteur.

Health Canada Grants Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People Ages 12 and Older Who Have at Least One F508del Mutation

Retrieved on: 
Friday, June 18, 2021
Research, Genetics, Clinical trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Cystic fibrosis, Drugs, Health sciences, Orphan drugs, Health care, Breakthrough therapy, Combination drugs, Cystic fibrosis transmembrane conductance regulator, Elexacaftor/tezacaftor/ivacaftor, Reshma Kewalramani, Vertex, Vertex Pharmaceuticals, TRIKAFTA®, Cystic Fibrosis, Vertex, TRIKAFTA®, CYSTIC FIBROSIS, VERTEX

With this approval, for the first time, approximately 1,100 eligible patients with CF ages 12 years and older who have at least one F508del mutation have a medicine that targets the underlying cause of their CF.

Key Points: 
  • With this approval, for the first time, approximately 1,100 eligible patients with CF ages 12 years and older who have at least one F508del mutation have a medicine that targets the underlying cause of their CF.
  • The approval of TRIKAFTA marks a significant milestone for Canadians with CF, their families and Vertex, said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex.
  • TRIKAFTA is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

ReCode Therapeutics Presents Preclinical Data Using SORT-LNP™ and RNA Platforms to Rescue CFTR Function at the 44th European Cystic Fibrosis Conference (ECFS)

Retrieved on: 
Friday, June 11, 2021
Science, Biotechnology, Research, Pharmaceutical, Health, Medical devices, Clinical trials, Other Health, Cystic fibrosis, Branches of biology, Medical specialties, Biology, Cystic fibrosis transmembrane conductance regulator, Batsheva Kerem, Eric Sorscher, Cystic Fibrosis, ReCode Therapeutics, CYSTIC FIBROSIS, RECODE THERAPEUTICS

These data showcase the potential of our differentiated RNA-based molecular therapies to treat cystic fibrosis.

Key Points: 
  • These data showcase the potential of our differentiated RNA-based molecular therapies to treat cystic fibrosis.
  • By leveraging our LNP platform to deliver genomic medicines, we have demonstrated the ability to restore cystic fibrosis transmembrane conductance regulator (CFTR) function through both tRNA and mRNA approaches, said David Lockhart, Ph.D., CEO & President, ReCode Therapeutics.
  • RCT223 was shown to restore CFTR function for at least 72 hours in patient-derived hBE cells after a single administration.
  • The Companys pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia.