Cystic fibrosis

JJP Biologics Announces Positive Decision to Execute a First-in-Human Clinical Trial of Anti-Inflammatory mAb JJP-1212 (anti-CD89)

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星期三, 五月 29, 2024
Biotechnology, General Health, Health, Pharmaceutical, Clinical Trials, JJP, Patient, EudraCT, Biology, Blister, Immunoglobulin G, European Medicines Agency, Clinical trial, Linear IgA bullous dermatosis, Doctor of Philosophy, Immunoglobulin M, Trial of the century, Autoimmunity, Disease, Safety, Henoch–Schönlein purpura, Autoantibody, Fatty liver disease, IgA nephropathy, Trust, Civil service commission, European Commission, Idiopathic pulmonary fibrosis, Cancer, CSO, Axial spondyloarthritis, Lupus, Biomarker, Incidence, Chronic obstructive pulmonary disease, Volunteering, Cystic fibrosis, Hidradenitis suppurativa, Linearity, European, Paratriathlon, Rheumatoid arthritis, Immunoglobulin A, Pharmaceutical industry

Up to date, JJP Biologics is the first Polish company to receive approval to perform the First-in-Human clinical trial with a novel therapeutic monoclonal antibody.

Key Points: 
  • Up to date, JJP Biologics is the first Polish company to receive approval to perform the First-in-Human clinical trial with a novel therapeutic monoclonal antibody.
  • According to Paweł Szczepański, COO and Management Board Member at JJP Biologics - “This is a historically unprecedented approval of a first-in-human clinical trial for a novel large molecule therapy from Poland.
  • JJP Biologics is exploring the development of companion diagnostics in various indications using serum IgA autoantibodies as biomarkers for personalized treatment with JJP-1212.
  • JJP Biologics pursues the development of its product candidates as well as projects executed in cooperation with scientific partners.

Arcturus Therapeutics Announces Positive Development for Cystic Fibrosis Program

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星期二, 五月 28, 2024
Biotechnology, Health, General Health, Pharmaceutical, Health Technology, Research, Infectious Diseases, Genetics, Science, COVID-19, Clinical Trials, Patient, Liver, Messenger, Therapy, European, Conference, Cystic fibrosis, Spirometry, RNA, Arcturus Therapeutics, ARCT, FEV1, Pharmaceutical industry

Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and medicines to treat unmet medical needs within liver and respiratory rare diseases, today announced Arcturus will be presenting Phase 1 results in healthy volunteers and new Phase 1b interim data in CF patients for ARCT-032, an inhaled investigational mRNA therapeutic to treat CF, at the 47th European Cystic Fibrosis Conference on June 7, 2024.

Key Points: 
  • Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and medicines to treat unmet medical needs within liver and respiratory rare diseases, today announced Arcturus will be presenting Phase 1 results in healthy volunteers and new Phase 1b interim data in CF patients for ARCT-032, an inhaled investigational mRNA therapeutic to treat CF, at the 47th European Cystic Fibrosis Conference on June 7, 2024.
  • “We were pleased to observe ARCT-032 treatments for the first four patients were generally safe and well tolerated with no serious adverse events,” said Joseph Payne, President & CEO of Arcturus Therapeutics.
  • “Furthermore, we observed an encouraging trend towards lung function improvements in the first four CF participants after only two inhaled administrations.”
    “The absolute change in percent predicted FEV1 (Forced Expiratory Volume in 1 second) in the first four CF participants was observed to be an average of 4.0% at day 8,” said Dr. Juergen Froehlich, Chief Medical Officer of Arcturus Therapeutics.
  • “I am pleased to announce our team will present this new Phase 1b interim data at the upcoming European CF conference in Scotland, supplementing the favorable results from our Phase 1 single ascending dose study in healthy volunteers.”

Wakefern Food Corp. Donates $1 Million to Kean University for Scholarship Honoring Alumnus Joe Sheridan

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星期二, 五月 21, 2024
Fairway Market, Business, Social responsibility, Supermarket, Kean University, Science, Education, The Fresh Grocer, Cystic Fibrosis Foundation, Dream, Cystic fibrosis, GPA, CBPM, Student, Gourmet Garage, ShopRite

Union, N.J., May 21, 2024 (GLOBE NEWSWIRE) -- Wakefern Food Corp. has announced a $1 million donation for student scholarships at Kean University in honor of the company’s recently-retired president, Kean alumnus Joe Sheridan ’80.

Key Points: 
  • Union, N.J., May 21, 2024 (GLOBE NEWSWIRE) -- Wakefern Food Corp. has announced a $1 million donation for student scholarships at Kean University in honor of the company’s recently-retired president, Kean alumnus Joe Sheridan ’80.
  • “Wakefern’s generous gift honoring Kean alumnus Joe Sheridan creates a lasting legacy at Kean and offers students a financial safety net to continue their studies and follow in the footsteps of Joe and other prominent Kean alumni,” said Kean President Lamont O. Repollet, Ed.D.
  • Sheridan said he was grateful for the education he received at Kean, which is New Jersey’s urban research university.
  • Creating the scholarship at Kean University was a fitting way to honor his legacy at Wakefern.

BiomX Reports First Quarter 2024 Financial Results and Provides Business and Program Updates

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星期二, 五月 21, 2024
Patient, Infection, Staphylococcus aureus, ESCMID, Pseudomonas aeruginosa, Therapy, APT, NESS, Orphan, OrbiMed, Cystic Fibrosis Foundation, Cystic fibrosis, DFO, S. aureus, Șaeș, Abstract, Part, Phage therapy, FEV1, Pharmaceutical industry

GAITHERSBURG, Md. and NESS ZIONA, Israel, May 21, 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 reported financial results and provided business and program updates for the first quarter ended March 31, 2024. BiomX is announcing merged financial reporting for the first time following the closing of its merger with Adaptive Phage Therapeutics, Inc. (“APT”) in March 2024 and a concurrent $50 million private placement.

Key Points: 
  • and NESS ZIONA, Israel, May 21, 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 reported financial results and provided business and program updates for the first quarter ended March 31, 2024.
  • General and administrative expenses were $2.7 million for the first quarter of 2024, compared to $1.6 million for the first quarter of 2023.
  • Net loss was $17.3 million for the first quarter of 2024, compared to $6.4 million for the first quarter of 2023.
  • ET to discuss its first quarter 2024 financial results and to provide a corporate update.

Krystal Biotech to Present at the American Thoracic Society 2024 International Conference

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星期三, 五月 15, 2024
Lung, Patient, CFTR, American Thoracic Society, AATD, Treatment, Conference, KRYS, Cystic fibrosis, Gene, ATS, Mutation

PITTSBURGH, May 15, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, announced today that the Company will be presenting new preclinical data on respiratory genetic medicine candidates KB407 and KB408 at the American Thoracic Society (ATS) 2024 International Conference being held from May 17-22, 2024 in San Diego, California.

Key Points: 
  • PITTSBURGH, May 15, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, announced today that the Company will be presenting new preclinical data on respiratory genetic medicine candidates KB407 and KB408 at the American Thoracic Society (ATS) 2024 International Conference being held from May 17-22, 2024 in San Diego, California.
  • KB407 and KB408 are modified, replication-defective, non-integrating HSV-1 vectors formulated for inhaled delivery to the lung via nebulization.
  • KB408 encodes two full-length copies of alpha-1 antitrypsin and is under investigation for the treatment of alpha-1 antitrypsin deficiency (AATD).
  • Following the presentation, the posters will also be available to view online on the Investor section of the Company’s website .

Sound Pharmaceuticals’ IND Involving Cochlear Implantation Allowed by the FDA

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星期一, 五月 20, 2024
Biotechnology, Consumer Electronics, Technology, FDA, People with Disabilities, Health, Pharmaceutical, Medical Devices, Audio, Video, Consumer, Clinical Trials, Patient, Ototoxicity, Dental implant, Sensorineural hearing loss, MED-EL, Risk, SPI, IND, Dizziness, Safety, Principal investigator, MD, Cystic Fibrosis Foundation, Cystic fibrosis, Growth, Ingeborg, Hearing loss, Fast Track, Ebselen, Communication, Vertigo, Glutathione peroxidase, Quality of life, NIH, Hearing, Tinnitus, Pharmaceutical industry

Sound Pharmaceuticals (SPI) is pleased to announce that it has received FDA notification that its Investigational New Drug Application (IND) may proceed involving SPI-1005 treatment and cochlear implantation (CI).

Key Points: 
  • Sound Pharmaceuticals (SPI) is pleased to announce that it has received FDA notification that its Investigational New Drug Application (IND) may proceed involving SPI-1005 treatment and cochlear implantation (CI).
  • The Phase 2 trial will enroll 40 adults who are CI candidates and scheduled to receive a cochlear implant.
  • This marks the 6th IND involving SPI-1005, which has been tested in 13 studies enrolling over 850 participants to date.
  • SPI and MED-EL entered into an exclusive worldwide collaborative agreement involving the use of SPI-1005 with cochlear implants.

Jennifer Schneider Elected to Vertex Board of Directors

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星期三, 五月 15, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Diabetes, Patient, Biology, Hospital, College, Obesity, Stanford University, Internal medicine, Sickle cell disease, Population health, Castlight Health, Vertex Pharmaceuticals, Hypertension, Research, Policy, Woman, Teladoc Health, Cystic fibrosis, Beta thalassemia, VRTX, Holy Cross, History

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Jennifer Schneider, M.D., M.S., has been elected to its Board of Directors as an independent director.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Jennifer Schneider, M.D., M.S., has been elected to its Board of Directors as an independent director.
  • Dr. Schneider has more than two decades of experience in the health care industry as a physician, scientist and health care executive.
  • She currently serves on the Board of Directors of Jasper Health and Maven Health.
  • The addition of Dr. Schneider brings Vertex’s board of directors to 11 members, nine of whom are independent and 45% of whom are women.

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.