Chronic granulomatous disease

Prime Medicine Reports Second Quarter 2023 Financial Results and Provides Business Updates

Retrieved on: 
Monday, August 7, 2023
Myelodysplastic syndrome, Exercise, Chronic granulomatous disease, Partnership, Gene, SCIP, Eye, Immunotherapy, G&A, Toxicity, Research, Economics, CD34, Acute myeloid leukemia, Transplant, MDS, Health, AML, Investment, Patient, Cancer, Annual general meeting, Cell, CGD, American Society of Gene and Cell Therapy, Liver, Prime editing, HSC, R, Therapy, CD117, Society, DSM-IV codes, D, Medical device, Pharmaceutical industry, Vaccine, Video game, Cryptocurrency, IND

CAMBRIDGE, Mass., Aug. 07, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided business updates for the second quarter ended June 30, 2023.

Key Points: 
  • In May 2023, Prime Medicine presented new preclinical data at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Initiate investigational new drug (IND)-enabling studies for PM359 in CGD in 2023.
  • Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.
  • Second Quarter 2023 Financial Results:
    R&D Expenses: Research and development (R&D) expenses were $34.6 million for the three months ended June 30, 2023, as compared to $18.9 million for the three months ended June 30, 2022.

Prime Medicine to Present New Preclinical Data Highlighting Broad Potential of Prime Editing Technology at ASGCT 26th Annual Meeting

Retrieved on: 
Wednesday, May 17, 2023
Chronic granulomatous disease, DNA, TRAC, Gene, CD19, Neoplasm, Bone marrow, Investigational New Drug, Deletion, Fungus, CD34, Stem cell, Health, Chromosomal translocation, Spleen, Genome, NADPH, Annual general meeting, Cancer, Cell, CGD, American Society of Gene and Cell Therapy, Society, Disease, NCF1, Vaccine, B2M

CAMBRIDGE, Mass., May 17, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced the presentation of new preclinical data that further demonstrated the potential of Prime Editing to correct the causative mutation of chronic granulomatous disease (CGD) and preclinical data that showcased the potential application of the Prime Editing Assisted Site-Specific Integrase Gene Editing (PASSIGE™) platform to generate multiplex-edited CAR-T cells for the treatment of certain cancers and immune diseases. The data are being presented today during the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023, in Los Angeles, California.

Key Points: 
  • The data are being presented today during the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20, 2023, in Los Angeles, California.
  • “We are very pleased to present these new data for our CGD program and PASSIGE platform today at ASGCT, which underscore our belief in the breadth and potential of Prime Editing to offer curative treatments for many diseases,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine.
  • Further, while the benefits of autologous CAR-T therapies are well established, their full potential is often hindered by manufacturing and delivery challenges.
  • Prime Medicine has previously shared data from the CGD program that demonstrated the ability of Prime Editing to correct a CGD causative mutation in CD34+ cells ex vivo.

Prime Medicine Reports First Quarter 2023 Financial Results and Provides Business Updates

Retrieved on: 
Thursday, May 11, 2023
Spark Therapeutics, Chronic granulomatous disease, Intellectual property, Patent, Pfizer, Bleeding, Gene, CHOP, Eye, Food and Drug Administration, RNA, G&A, Retina, Sale, Novartis, Investment, Patient, Cancer, Annual general meeting, Cell, CGD, American Society of Gene and Cell Therapy, Liver, Prime editing, R, Research, Therapy, Society, Food, DSM-IV codes, D, Management, Cryptocurrency, Pharmaceutical industry, Vaccine, Video game, Spark, IND

CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided business updates for the first quarter ended March 31, 2023.

Key Points: 
  • 17/751,599 --
    -- Cash, cash equivalents, investments and restricted cash balance of $263.0 million as of March 31, 2023 --
    CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided business updates for the first quarter ended March 31, 2023.
  • In April 2023, Prime Medicine received a notice of allowance from the U.S. PTO for its third patent application, No.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Initiate investigational new drug (IND)-enabling studies for PM359 in CGD in 2023.
  • Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.

Prime Medicine Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 9, 2023
LNP, GAA, FXN, CMC, AAV, Chemistry, R, CF, Quarter, Stem cell, Administration, Patient, Chronic granulomatous disease, Mitochondrion, Eye, HSC, CFTR, Research and development, Research, Central nervous system, Liver, Swelling, Gene expression, Investment, Cell, WD, Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, FRDA, CGD, Pharmaceutical industry, Vaccine, Video game, Cryptocurrency, IND

CAMBRIDGE, Mass., March 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2022.

Key Points: 
  • “In 2022, we made important progress in our efforts to build Prime Medicine and demonstrate the promise of Prime Editors as a potentially best-in-class genetic medicine approach,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Initiate investigational new drug (IND)-enabling studies of PM359 for the treatment of CGD in 2023.
  • Maximize Prime Editing’s broad therapeutic potential and create value through strategic business development that extends the reach and impact of Prime Editing to areas beyond Prime Medicine’s current areas of focus.
  • Fourth Quarter and Full Year 2022 Financial Results:
    Research and Development (R&D) Expenses: R&D expenses were $29.1 million for the fourth quarter of 2022 and $86.7 million for the year ended December 31, 2022, as compared to $52.9 million for the fourth quarter of 2021 and $70.6 million for the year ended December 31, 2021.

Prime Medicine Announces Recent Progress and Highlights 2023 Strategic Priorities

Retrieved on: 
Monday, January 9, 2023
Disease, FRDA, Genome, Cystic fibrosis transmembrane conductance regulator, Swelling, CGD, Safety data sheet, Respiratory tract, GAA, Tissue, Cystic fibrosis, CD34, CMC, AAV, Heart, Failure rate, CFTR, Mitochondrion, Culture, DNA, LNP, Safety, Mutation, IPSC, Gene editing, Central nervous system, Hepatocyte, Patient, Degenerative disease, CF, Bronchiole, Neuron, PCSK9, Ataxia, Liver, Human, FXN, Inflammation, Chronic granulomatous disease, Eye, Investment, Prime editing, Vaccine, Pharmaceutical industry, Medical device, IND, WD, Frataxin

CAMBRIDGE, Mass., Jan. 09, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today provided an update on recent advancements of its Prime Editing technology and progress across its initial pipeline of eighteen programs, and outlined its strategic priorities and expected milestones for 2023.

Key Points: 
  • Today, Prime Medicine announced new preclinical data demonstrating that Prime Editing-mediated removal of pathological repeats in vitro results in correction of hypermethylation at the FXN gene, restoring genetic function back to wild-type levels.
  • Off-target Safety Data: Prime Medicine is progressing a comprehensive suite of assays to evaluate the potential off-target activity of its Prime Editors.
  • Prime Medicine is using PCSK9 as a model system for developing its modular LNP delivery platform to the liver.
  • Prime Medicine expects the following activities and next steps to drive the Prime Editing platform forward:
    Nominate first development candidate for CGD in 1Q 2023.

STACHES TO LASHES

Retrieved on: 
Thursday, September 15, 2022
National, MD, Sohei Nihi, National Institutes of Health Clinical Center, The Front Porch, No Place Like Home (Buffy the Vampire Slayer), Children, Chronic granulomatous disease, Family, Restaurant, Research, DNA, NIH, Immunodeficiency, Pharmaceutical industry, Child

BETHESDA, Md., Sept. 15, 2022 /PRNewswire/ -- The Children's Inn at the National Institutes of Health (NIH) "Staches to Lashes" a benefit for The Children's Inn at The National Institutes of Health (NIH), to be held on October 20, 2022 at The Cliff House Resort in Cape Neddick, Maine.

Key Points: 
  • "I initiated this event in 2017 during my first summer as the owner of The Front Porch.
  • Over the first three years of this event, we went from raising $32,000 in 2017 to $137,000 in 2019," said Vogel.
  • 'Staches to Lashes' is a very special one; for which I have been humbled to receive so much support."
  • For more information about Staches to Lashes, please call 207-646-4005 or visit thefrontporch.com.

Henkel Corp. Voluntarily Recalls Alterna® Bond Repair Leave-in Heat Protection Spray due to Possible Health Risk

Retrieved on: 
Friday, May 27, 2022
Cosmetics, Retail, Risk, Knowledge, Chronic lung disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Infection, Chronic granulomatous disease, Burkholderia cepacia complex, Corporation, Degenerative disease, Multimedia, Cystic fibrosis, Patient, Henkel North American Consumer Goods, Sale, Henkel, Dietary supplement, BCC

Patients with underlying lung disease (especially cystic fibrosis) might be at increased risk for severe infections with Bcc.

Key Points: 
  • Patients with underlying lung disease (especially cystic fibrosis) might be at increased risk for severe infections with Bcc.
  • View the full release here: https://www.businesswire.com/news/home/20220526005797/en/
    The potential for product contamination was noted after testing performed by Henkel revealed the presence of Bcc in some 4.2 ounce bottles of Alterna Bond Repair Leave-in Heat Protection Spray.
  • While Henkel has received no reports of harm or injury in connection with this product, the company is proactively recalling the product.
  • The Alterna Bond Repair Leave-in Heat Protection Spray included in this recall comes in a 4.2 ounce, purple plastic spray bottle marked with lot # Y401427403 on the bottom, and was distributed nationwide through a distributor.

Jasper Therapeutics Announces Treatment of First Patient in Study of JSP191 Conditioning in Patients with Fanconi Anemia

Retrieved on: 
Tuesday, May 17, 2022
FA, Physician, Cancer, B cell, Stem cell, Risk, Company, Population, Aplastic anemia, Blood, RNA transfection, Medicine, Stanford University School of Medicine, MDS, Chronic granulomatous disease, HSCT, HSC, Bone marrow failure, Degenerative disease, AML, Patient, Biology, Sickle cell disease, NASDAQ, Bone marrow, Therapy, Fanconi anemia, COVID-19, GATA2, SCID, Microdeletion syndrome, Research, Definitive, Lists of diseases, Anemia, Probability, Center, Toxicity, Forward-looking statement, Transplant, Safety, Immune reconstitution inflammatory syndrome, Private Securities Litigation Reform Act, SEC, MD, CEO, GLOBE, Pharmaceutical industry, Vaccine, Jasper, Pediatrics

Fanconi Anemia (FA) is a rare but serious blood disorder that prevents the bone marrow from making sufficient new red blood cells.

Key Points: 
  • Fanconi Anemia (FA) is a rare but serious blood disorder that prevents the bone marrow from making sufficient new red blood cells.
  • Treatment may include blood transfusions or medicine to create more red blood cells, but hematopoietic stem cell transplant (HSCT) is the only cure.
  • Primary outcome measures include the number of patients without treatment emergent adverse events following administration of JSP191.
  • While Jasper may elect to update these forward-looking statements at some point in the future, Jasper specifically disclaims any obligation to do so.

Jasper Therapeutics to Present Updated Data on JSP191 Conditioning in SCID Patients at the 2022 Clinical Immunology Society Annual Meeting

Retrieved on: 
Thursday, March 31, 2022
Company, Risk, Therapy, Life, Stem cell, Population, NASDAQ, Degenerative disease, Translational medicine, AML, SCID, Forward-looking statement, GATA2, IVIG, Bone marrow, SEC, Probability, CEO, Senior, Private Securities Litigation Reform Act, HSC, MDS, Lists of diseases, CIS, Safety, Biology, Annual general meeting, RNA transfection, Microdeletion syndrome, COVID-19, GLOBE, Immune reconstitution inflammatory syndrome, Transplant, Severe combined immunodeficiency, Sickle cell disease, Physician, Fanconi anemia, Chronic granulomatous disease, Pharmaceutical industry, Vaccine, Jasper, Research, Patient, MD

JSP191 based conditioning may provide these patients with the best chance of a safe and successful transplant and reconstituted immune system.

Key Points: 
  • JSP191 based conditioning may provide these patients with the best chance of a safe and successful transplant and reconstituted immune system.
  • CIS attendees are the primary caregivers for the immune deficient patient population, we are pleased to be able to present this data at the 2022 CIS annual meeting, Ronald Martell, CEO of Jasper.
  • We believe that with our successful clinical efforts, we are one step closer, and uniquely positioned to deliver a targeted non-genotoxic conditioning agent to patients with SCID.
  • Three clinical trials for myelodysplastic syndromes (MDS)/acute myeloid leukemia (AML), severe combined immunodeficiency (SCID) and Fanconi anemia are currently enrolling.

Jasper Therapeutics Announces Research Collaboration with AVROBIO to Evaluate JSP191 as Conditioning Agent in Clinical Studies of Ex Vivo Lentiviral Gene Therapy

Retrieved on: 
Tuesday, November 9, 2021
Stem cell, Safety, AML, Bone marrow, Private Securities Litigation Reform Act, Therapy, Gaucher, Lists of diseases, Microdeletion syndrome, SCID, Prospectus, Lymphatic system, Security (finance), Autoimmune disease, MDS, Fabry disease, COVID-19, Fanconi anemia, Securities Act of 1933, Forward-looking statement, Jasper, Transplant, U.S. Securities and Exchange Commission, GLOBE, Sickle cell disease, Degenerative disease, SEC, Chronic granulomatous disease, Drug Quality and Security Act, GATA2, Biology, Patient, Risk, Pharmaceutical industry, Vaccine

This collaboration with AVROBIO further expands the potential of JSP191 for use with lentiviral gene therapies, said Bill Lis, executive chairman and chief executive officer of Jasper Therapeutics.

Key Points: 
  • This collaboration with AVROBIO further expands the potential of JSP191 for use with lentiviral gene therapies, said Bill Lis, executive chairman and chief executive officer of Jasper Therapeutics.
  • Jasper is focused on building a leading hematopoietic stem cell therapeutics company with the potential to bring one-time treatments to patients with blood cancers, rare monogenic diseases and autoimmune diseases.
  • Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell.
  • JSP191, an anti-CD117 monoclonal antibody, is in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplantation.