Cystic fibrosis

Royalty Pharma Reports First Quarter 2024 Results

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星期四, 五月 9, 2024
FDA, Ulcerative colitis, KarXT, Food, MorphoSys, European Commission, Marketing, New Drug Application, Cystic fibrosis, PDSS, PANSS, Tourette syndrome, Lupus, Docetaxel, Roche, Schizophrenia, Novartis, ADP, Pfizer, Incidence, Bristol Myers Squibb, Patient, Neurology, Acquisition, Sacituzumab govitecan, Olanzapine, Civil service commission, Multiple sclerosis, Positive, Vertex, Sanofi, Teva Pharmaceuticals, Astellas Pharma, European Medicines Agency, Lung cancer, Pharmaceutical industry

During 2024, Royalty Pharma announced new transactions of up to $619 million.

Key Points: 
  • During 2024, Royalty Pharma announced new transactions of up to $619 million.
  • In January 2024, Royalty Pharma acquired a royalty interest in ecopipam for an upfront payment of $49 million and up to $44 million in milestone payments contingent on the achievement of certain regulatory milestones.
  • In May 2024, Royalty Pharma announced a transaction to acquire royalties and milestones on frexalimab owned by ImmuNext for approximately $525 million in cash including estimated transaction costs.
  • Royalty Pharma has provided guidance for full year 2024, excluding transactions and borrowings announced after the date of this release, as follows:

4DMT Reports First Quarter 2024 Financial Results and Operational Highlights

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星期四, 五月 9, 2024
FDA, Regenerative medicine, Research, Choroideremia, Biopsy, European Medicines Agency, Injection, Overalls, Inflammation, Food, Alpha-1 antitrypsin, Cystic fibrosis, Chelsea Handler, Intravitreal administration, Conference, Retina, Atypical hemolytic uremic syndrome, DME, Angiogenesis, PRISM, AMD, Safety, Security (finance), Cash, Retinitis pigmentosa, Exercise, PRIME, EMA, NHP, Clinical trial, A1AT, ASRS, ARVO, ECFS, Șaeș, Cubic foot, Patient, Society, Geographic atrophy, Pharmaceutical industry

“The first quarter of 2024 kicks off another transformative year for 4DMT with exceptional progress across our product pipeline and platform in multiple therapeutic areas, particularly our lead program 4D-150 in large market VEGF-driven retinal diseases,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.

Key Points: 
  • “The first quarter of 2024 kicks off another transformative year for 4DMT with exceptional progress across our product pipeline and platform in multiple therapeutic areas, particularly our lead program 4D-150 in large market VEGF-driven retinal diseases,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • R&D Expenses: Research and development expenses were $27.9 million for the first quarter of 2024, as compared to $22.4 million for the first quarter of 2023.
  • G&A Expenses: General and administrative expenses were $10.3 million for the first quarter of 2024, as compared to $8.0 million for the first quarter of 2023.
  • Net Loss: Net loss was $32.4 million for the first quarter of 2024, as compared to net loss of $28.7 million for the first quarter of 2023.

Intellia Therapeutics Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress

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星期四, 五月 9, 2024
FDA, Bone marrow, CRISPR, Research, Recode, IND, KLKB1, Medicine, Polyneuropathy, NK, AATD, Corporation, Congress, Brain, TTR, Amyloidosis, Food, Gene editing, Conference, Eye, Corporate social responsibility, BLA, HAE, Therapy, Safety, Tissue, Cystic fibrosis, Cardiomyopathy, ATM, DNA, Intellia Therapeutics, Bank, EAACI, Lung, AAT, Social, ATTR, NTLA, Cell, Disease, Regeneron Pharmaceuticals, Patient, Liver, ESG, Pharmaceutical industry

“Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies.

Key Points: 
  • “Intellia continues to make outstanding progress across our pipeline of in vivo and ex vivo single-dose CRISPR-based therapies.
  • Additionally, Intellia plans to report topline results from the Phase 2 portion in mid-2024 and present full results at a medical meeting in the second half of 2024.
  • Collaboration Revenue: Collaboration revenue was $28.9 million during the first quarter of 2024, compared to $12.6 million during the first quarter of 2023.
  • Net Loss: Net loss was $107.4 million for the first quarter of 2024, compared to $103.1 million during the first quarter of 2023.

Polarean’s Xenon MRI to be Featured at Upcoming ATS 2024 Conference

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星期三, 五月 8, 2024
Hypoxemia, Supplement, Pulmonology, Patient, Adolescence, Inhalation, ATS, Euphoria, Adrenal insufficiency, Cystic fibrosis, Dizziness, AIM, Oxygen saturation (medicine), Doctor of Philosophy, Conference, Research, Bronchopulmonary dysplasia, Radiation-induced lung injury, Heart rate, Ageing, Transience, Headache, RIS, MRI, Magnetic resonance imaging, Hypoesthesia, Government, Asthma, Risk, COVID-19, Orthostatic hypertension, COPD, Medical imaging

The summit is scheduled to take place on May 17th-18th in San Diego, CA, at the Manchester Grand Hyatt San Diego.

Key Points: 
  • The summit is scheduled to take place on May 17th-18th in San Diego, CA, at the Manchester Grand Hyatt San Diego.
  • The Respiratory Innovation Summit, a premier event in the field of pulmonary medicine, unites innovators, investors, clinicians, and advocacy groups.
  • After the dynamic discussions of the Respiratory Innovation Summit, Polarean eagerly anticipates further collaboration within the pulmonary medicine community at the 2024 ATS International Conference, held from May 19th to 22nd.
  • Christopher von Jako, PhD, CEO of Polarean, said: “The Respiratory Innovation Summit and the ATS annual conference serve as invaluable venues to spotlight our Xenon MRI platform, especially to those enthusiastic about fostering collaborative partnerships.

Global Nebulizers Research Report 2024: Market to Reach $2.5 Billion by 2032 - Product Type, Portability, End-User, Application Analysis - ResearchAndMarkets.com

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星期四, 五月 9, 2024
Medical Devices, Health, Hospital, Prognosis, Tobacco, Cystic fibrosis, Incidence, Asthma, Organization, COPD, Pharmaceutical industry

The "Global Nebulizers Market Report by Product Type, Portability, End-User, Application, and Region 2024-2032" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Nebulizers Market Report by Product Type, Portability, End-User, Application, and Region 2024-2032" report has been added to ResearchAndMarkets.com's offering.
  • The global nebulizers market is witnessing an impressive growth trajectory, with projections indicating a market value of US$ 2.5 Billion by the year 2032.
  • The global nebulizers market report meticulously segments the market by product type, portability, end-user, and application, presenting an in-depth analysis of each sector and its projected growth.
  • The global nebulizers market is on a steady growth path and encapsulates significant opportunities across varied sectors.

Arcturus Therapeutics Announces First Quarter 2024 Financial Update and Pipeline Progress

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星期三, 五月 8, 2024
Biotechnology, Infectious Diseases, COVID-19, Health, Pharmaceutical, Society, COVID, Investment, Meiji, ARCT, Ornithine transcarbamylase deficiency, Safety, SARS-CoV-2, Travel, European Commission, Research, OTC, Cystic fibrosis, Lyme disease, RNA, MAA, Inborn errors of metabolism, Trial of the century, Vaccine, CDMO, Omicron, SRR, GMT, SAD, Liver, CSL, EMA, JPMorgan Chase, SIMD, Civil service commission, Infection, Messenger, Arcturus Therapeutics, Therapy, European Medicines Agency, Pharmaceutical industry

“Arcturus continues to make encouraging progress in both our vaccine and therapeutics pipeline,” said Joseph Payne, President & CEO of Arcturus.

Key Points: 
  • “Arcturus continues to make encouraging progress in both our vaccine and therapeutics pipeline,” said Joseph Payne, President & CEO of Arcturus.
  • To support this effort, Arcturus along with CDMO partners are on track to deliver the initial 4 million commercial doses of Kostaive in Q3.
  • The Company will share a progress update on the Phase 2 study on July 1, 2024.
  • The expected cash runway extends at least three years based on the current pipeline and programs through the first quarter of fiscal year 2027.

Getting the Math Right: The Growing Movement to Improve How Medicines Are Valued Will Take Center Stage at ISPOR 2024

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星期五, 五月 3, 2024
Negotiation, Cancer, Bias, Hospital, Policy, Impact, Economics, Health, Cystic fibrosis, Generalization, Thought, Innovation, Doctor of Philosophy, Research, Literature, Elasticity, ICER, Animation, Federation, Organization, Patient, Medicine, Movement, Inflation, Medicare, CEA, Value, Professional, Pharmaceutical industry

"For too long, health plans and foreign governments have relied on faulty, outdated, and biased math that undervalues the benefits of lifesaving medicines to society.

Key Points: 
  • "For too long, health plans and foreign governments have relied on faulty, outdated, and biased math that undervalues the benefits of lifesaving medicines to society.
  • Several states also are considering adopting conventional CEA through so-called Prescription Drug Affordability Boards.
  • It will also preserve society’s incentives for the development of the next generation of innovative and valuable medicines.
  • This journey is particularly recommended for health economists who work in industry and those who work on projects for industry.

Carbon Biosciences Announces Four Presentations at the American Society of Gene and Cell Therapy 27th Annual Meeting

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星期四, 五月 2, 2024
Carbon, Parvoviridae, Analytic, Tropism, Poster, Heart, Cystic fibrosis, CFTR, Primate, Cell, Abstract, AAV, PAVE, Liver, Patient, Gene, Process, Society, Lung, Annual general meeting, Capsid, CBN, Knowledge, Vaccine

WALTHAM, Mass., May 2, 2024 /PRNewswire/ -- Carbon Biosciences, a preclinical stage biotechnology company developing genetic medicines for the treatment of pulmonary and cardiac diseases, announced that it has been selected for an oral presentation at the Presidential Symposium during the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place from May 7-11, 2024, in Baltimore, MD. The presentation will highlight the development of CGT-001, a potential gene therapy for the treatment of Cystic Fibrosis (CF), which utilizes the novel pulmonary targeting vector, CBN-1000, capable of delivering genes beyond the capacity of AAV. In addition, the Company will be featuring three poster presentations detailing new preclinical data, and manufacturing process development and analytical characterization for two of its novel viral gene therapy vectors.

Key Points: 
  • In addition, the Company will be featuring three poster presentations detailing new preclinical data, and manufacturing process development and analytical characterization for two of its novel viral gene therapy vectors.
  • Key insights from Carbon Biosciences' presentations at ASGCT:
    CBN-1000, a non-AAV parvovirus-based gene therapy vector, demonstrates excellent tolerability and transgene expression throughout the lung and airway in nonhuman primates after nebulization.
  • CBN-1100 vector demonstrates transgene packaging of up to 5.9 kb, representing >1 kb more capacity than AAV vectors.
  • The complete absence of expression in the liver suggests the potential for a differentiated specificity and safety profile compared to AAV-based vectors for cardiac gene therapy.

BiomX to Present Data from Phase 1b/2a Study of BX004 for the Treatment of Cystic Fibrosis Patients with Chronic Pulmonary Infections at the 34th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID 2024)

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星期二, 四月 23, 2024
NESS, Abstract, Cystic fibrosis, ECCMID, Patient

CAMBRIDGE, Mass.

Key Points: 
  • CAMBRIDGE, Mass.
  • and NESS ZIONA, Israel, April 23, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX” or the “Company”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced the Company will present data from the Phase 1b/2a study of BX004 for the treatment of cystic fibrosis patients with chronic Pseudomonas aeruginosa pulmonary infections at ECCMID 2024, which is being held from 27-30 April 2024, in Barcelona, Spain.
  • The abstract submitted by the Company was selected as a “Top Poster”, ranking it among the 1-2% of top-rated abstracts in the category submitted and accepted at ECCMID 2024.
  • Chronic Pseudomonas aeruginosa pulmonary infection treated with a nebulised phage cocktail in patients with cystic fibrosis: a phase 1b/2a randomised, double-blind, placebo-controlled, multicentre study

Krystal Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of Inhaled KB707 for the Treatment of Locally Advanced or Metastatic Solid Tumors of the Lung

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星期一, 四月 22, 2024
FDA, Cancer, Inhalation, Interleukin, Suma, Food, Cystic fibrosis, Drug, KRYS, NCT, Senior, Research and development, Patient, Lung, Clinical trial, Krystal, Gene, Pharmaceutical industry

PITTSBURGH, April 22, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, announced today that the first patient was dosed in its Phase 1 clinical trial (KYANITE-1) evaluating inhaled KB707, a modified HSV-1 vector designed to deliver genes encoding both human interleukin-12 (IL-12) and interleukin-2 (IL-2) to the lung, for the treatment of patients with locally advanced or metastatic solid tumors of the lung.

Key Points: 
  • “Cytokine delivery via inhalation is a first-of-its-kind therapeutic approach made possible by the unique attributes of Krystal’s HSV-1-based vector platform.
  • Together with intratumoral KB707, inhaled KB707 has the potential to significantly expand the clinical utility of cytokine therapy to treat a wide range of otherwise difficult-to-treat and standard of care refractory solid tumors.
  • “Dosing the first patient in our inhaled KB707 trial is another important milestone for our oncology program and for Krystal,” said Suma Krishnan, President, Research & Development, Krystal Biotech.
  • In July 2023, the FDA granted intratumoral KB707 Fast Track Designation for the treatment of anti-PD-1 relapsed/refractory locally advanced or metastatic melanoma.