Transferrin

Dyadic Reports 2023 Full Year Results and Recent Company Progress

Retrieved on: 
Thursday, March 28, 2024
Cygnus, Enzyme, Rabies, SAHPRA, Biology, Sale, Innovation, Infection, Note, European, AVATAR, Therapy, Host cell protein, Vaccine, Analysis, Convertible, Research, Biotechnology, A-DNA, Massachusetts General Hospital, Partnership, Melamaravakkadu, Webcast, Primate, RNA, Biofuel, C1, Virology, SME, Safety, Nature Communications, Transferrin, Livestock, Global health, Manuscript, Food, Senior, NIIMBL, Antibody, Hospital, Antigen, Diagnosis, Health, Pithing, RBD, Phibro Animal Health, COVID-19, Dyadic, Messenger, Protein, Human

“In 2023, Dyadic achieved significant milestones in unlocking the potential of its microbial platforms.

Key Points: 
  • “In 2023, Dyadic achieved significant milestones in unlocking the potential of its microbial platforms.
  • We believe that we are well-positioned, both financially and scientifically, to execute our strategic plan with enhanced capabilities and resources.
  • For the year ended December 31, 2023, the Company received a total of approximately $1.3 million in connection with the sale of Alphazyme LLC.
  • Other Income: For the year ended December 31, 2023, the Company had a gain of approximately $1,018,000 from the sale of the Company’s equity interest in Alphazyme, LLC.

Dyadic to Attend Future Food Tech Event March 21 – 22, 2024 in San Francisco, California

Retrieved on: 
Thursday, March 21, 2024
Transferrin, Biotechnology, Partnership, Business development, Dyadic, Food, San Francisco Marriott Marquis, Medium (website), Future, Vaccine

Dyadic will be showcasing its Dapibus™ Microbial Expression System and providing updates on the progress of current products under development within its alternative proteins business unit.

Key Points: 
  • Dyadic will be showcasing its Dapibus™ Microbial Expression System and providing updates on the progress of current products under development within its alternative proteins business unit.
  • We believe this system holds promise for revolutionizing protein expression and production in various industries, including food, nutrition, and healthcare.
  • Moreover, Dyadic has recently entered into a co-marketing partnership with Biftek, Inc. (“Biftek”).
  • If you would like to schedule a meeting with our management members at Future Food Tech in San Francisco, please contact Heidi Zosiak at [email protected] or stop by the Dyadic International, Inc. booth (#20).

PulseSight Therapeutics Launches to Advance Non-viral Gene Therapies with Disruptive Minimally-Invasive Delivery Technology for Severe Retinal Diseases

Retrieved on: 
Wednesday, February 28, 2024
Doctor of Philosophy, Decorin, Drug development, Sale, Iris (anatomy), Transferrin, Ciliary muscle, Geographic atrophy, Ranibizumab, Iron, Retinitis pigmentosa, Retina, CNV, Paratriathlon, Eye, Brolucizumab, VEGF, Therapy, AMD, Independent, DNA, Retinal pigment epithelium, KIP, Doctor of Pharmacy, Safety, Novartis, Oxidative stress, CSO, Conditional sentence, RPE, EMA, MD, FDA, SAS, Choroidal neovascularization, Cell, Human, Patient, Glaucoma, Gene expression

Unique proprietary non-viral gene therapy platform with minimally invasive delivery technology providing long lasting gene expression and favorable distribution in the retina.

Key Points: 
  • Unique proprietary non-viral gene therapy platform with minimally invasive delivery technology providing long lasting gene expression and favorable distribution in the retina.
  • A substantially de-risked platform and two first-in-class gene therapies heading to the clinic, having the potential to become future blockbusters.
  • PARIS, Feb. 28, 2024 (GLOBE NEWSWIRE) -- PulseSight Therapeutics SAS, an ophthalmology biotech company developing disruptive non-viral gene therapies with minimally-invasive delivery technology, launches today with seed finance from Pureos Bioventures and ND Capital.
  • PulseSight is currently raising a Series A financing round to advance its programs into clinical proof-of-concept.

Orphan designation: Humanised IgG1 monoclonal antibody against TfR1 conjugated to exon 44 specific phosphorodiamidate morpholino oligonucleotide via a non-cleavable linker Treatment of Duchenne muscular dystrophy, 13/10/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, Duchenne muscular dystrophy, Committee, Transferrin, Immunoglobulin G, EMA, Pharmaceutical industry

EU/3/23/2838 - orphan designation for treatment of Duchenne muscular dystrophy

Key Points: 
  • EU/3/23/2838 - orphan designation for treatment of Duchenne muscular dystrophy
    Humanised IgG1 monoclonal antibody against TfR1 conjugated to exon 44 specific phosphorodiamidate morpholino oligonucleotide via a non-cleavable linker
    OrphanHuman
    MWB Consulting
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Orphan designation: Humanised IgG1 kappa fragment antibody targeting TfR1 conjugated to P125 oligonucleotide Treatment of myotonic disorders, 22/05/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
Transferrin, Immunoglobulin G, COMP, IRIS, Myotonia, EMA, European Commission, Patient, Committee, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Humanised IgG1 kappa fragment antibody targeting TfR1 conjugated to P125 oligonucleotide
    - Intended use
    - Treatment of myotonic disorders
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2781
    - Date of designation
    - Sponsor
    Pharma Gateway AB
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Orphan designation: humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker treatment of facioscapulohumeral muscular dystrophy, 15/02/2023 Positive

Retrieved on: 
Saturday, January 20, 2024
COMP, Immunoglobulin G, DUX4, European Commission, Small RNA, Transferrin, EMA, Facioscapulohumeral muscular dystrophy, IRIS, Committee, Pharmaceutical industry

EU/3/23/2756 - orphan designation for treatment of facioscapulohumeral muscular dystrophy

Key Points: 
  • EU/3/23/2756 - orphan designation for treatment of facioscapulohumeral muscular dystrophy
    humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker
    OrphanHuman
    MWB Consulting
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Apertura Gene Therapy Unveils Novel Engineered AAV Capsids Targeting Human Transferrin Receptor 1 for Neurological Gene Therapy Delivery

Retrieved on: 
Thursday, January 4, 2024
Health, Neurology, Genetics, Research, Science, Pharmaceutical, Biotechnology, Liver, Traffic barrier, Broad Institute, Transferrin receptor 1, DSM-IV codes, BBB, Vector, MIT, Astrocyte, Brain, Transferrin, Endothelium, Gene expression, AAV, Engineering, CNS, Harvard University, Transport, Central nervous system, Iron, Patient, Psychiatry, Gene therapy, Neuron, Vaccine

Apertura Gene Therapy , a biotechnology company opening opportunities in genetic medicine for treating debilitating diseases with limited options, today unveiled its proprietary engineered Adeno-Associated Virus (AAV) capsids that bind to the human Transferrin Receptor 1 (TfR1).

Key Points: 
  • Apertura Gene Therapy , a biotechnology company opening opportunities in genetic medicine for treating debilitating diseases with limited options, today unveiled its proprietary engineered Adeno-Associated Virus (AAV) capsids that bind to the human Transferrin Receptor 1 (TfR1).
  • This transportation mechanism mediates crossing of the blood-brain barrier (BBB) to enable gene therapy delivery throughout the central nervous system (CNS).
  • “Our TfR1 capsids, with their well-characterized mechanism of action, offer the unique advantage of significantly reducing human translation risk – a major historical challenge of engineering novel AAV capsids.
  • Apertura is currently advancing two programs utilizing its TfR1 capsids for undisclosed neurologic conditions and is leveraging its platform technologies to engineer novel payloads to regulate genetic expression and develop additional AAV capsids targeting specific human receptors.

Keros Therapeutics Presents Clinical Trial and Preclinical Study Results from its KER-050 Program and Preclinical Data from its ALK2 Inhibitor Program at the 28th Annual Congress of the European Hematology Association

Retrieved on: 
Friday, June 9, 2023
NT, Red blood cell, Cohort, Bone marrow, Patient, Iron, Male, Nosebleed, Transferrin, Fatigue, Diet, Myocardial infarction, Diarrhea, TI, HTB, Adenine, Research, Injection site reaction, Hematology, Heart, Chronic kidney disease, Solar cycle 1, COVID-19, Hematologic disease, Transfusion-dependent anemia, IWG, Biomarker, Acute myeloid leukemia, Erythropoiesis, Spleen, Anemia, Iron overload, ALK2, Therapy, Haematopoiesis, RBC, Ruxolitinib, European Hematology Association, LTB, Hematocrit, Part, Inflammation, MDS, Chronic obstructive pulmonary disease, EHA, RS, Nausea, Periodic breathing, Mouse, AI, CKD, KROS, Pharmaceutical industry, Dietary supplement, Medical device, Medical imaging, Silviculture, Residential care, Copper, Palladium, MF

In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.

Key Points: 
  • In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.
  • Of these patients, 37 had completed at least 24 weeks of treatment or discontinued as of the data cut-off date (the “evaluated RP2D patients”).
  • Data for hematological response and markers of hematopoiesis were presented from exploratory analyses of these evaluated RP2D patients.
  • By targeting ALK2 inhibition to suppress hepcidin, RKER-216 increased iron availability for erythropoiesis and partially rescued anemia in CKD mice.

Dyne Therapeutics’ Preclinical Data Demonstrating FORCE™ Platform Delivery to CNS Featured in Oral Presentation at ASGCT Annual Meeting

Retrieved on: 
Wednesday, May 17, 2023
DYN, Central nervous system, Asian literature, Therapy, Gene, IV, DM1, Brain, Cardiac muscle, RNA, CNS, CTG, Patient, FORCE, Deficit spending, Cell, DMPK, ASO, Phenotype, Oligonucleotide, Society, Transferrin, Disease, Vaccine, Medical device, Dyne

WALTHAM, Mass., May 17, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, is delivering an oral presentation today at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles highlighting new preclinical data demonstrating the FORCE™ platform achieved delivery to the central nervous system (CNS) in non-human primates and robust pharmacological effects in the brain in a model of myotonic dystrophy type 1 (DM1).

Key Points: 
  • The presentation will be available in the Scientific Publications & Presentations section of Dyne’s website following the session.
  • The FORCE platform was designed to overcome the limitations of delivering oligonucleotide therapeutics to muscle tissue by leveraging transferrin receptor 1 (TfR1).
  • TfR1-mediated delivery also has been shown by the field to facilitate uptake of therapeutics by the CNS.
  • Highlights from the ASGCT data include:
    In NHPs, FORCE conjugate achieved superior delivery compared to unconjugated ASO when both were administered via IV.

Dyne Therapeutics Reports First Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Thursday, May 11, 2023
DYN, Muscular Dystrophy Association, Dystrophin, Duchenne muscular dystrophy, Therapy, Gene, DM1, Sarcolemma, Pharmacokinetics, American Academy, G&A, Oligonucleotide, Facial muscles, Biomarker, CNS, Sale, DMD, Patient, Enrollment, Episcopal conference, FORCE, Annual general meeting, Cell, MAD, R, Research, FDA, MDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Society, Conference, Food, Transferrin, Pharmaceutical industry, Medical imaging, Cryptocurrency, Brain, Fibrosis, Dyne

WALTHAM, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the first quarter of 2023 and business highlights.

Key Points: 
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the first quarter of 2023 and business highlights.
  • Research and development (R&D) expenses: R&D expenses were $37.5 million for the quarter ended March 31, 2023, compared to $28.2 million for the quarter ended March 31, 2022.
  • General and administrative (G&A) expenses: G&A expenses were $7.9 million for the quarter ended March 31, 2023, compared to $7.5 million for the quarter ended March 31, 2022.
  • Net loss: Net loss for the quarter ended March 31, 2023 was $44.2 million, or $0.78 per basic and diluted share.