DMPK

Design Therapeutics Announces First Quarter 2024 Financial Results and Highlights Upcoming Program Milestones

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수요일, 5월 8, 2024
Patient, Design, Research, DMPK, IND, Observational study, HD, GLP, FA, DM1, HTT, Therapy, Biotechnology, G&A, Pharmaceutical industry

CARLSBAD, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced first quarter 2024 financial results along with recent business highlights and upcoming milestones.

Key Points: 
  • Starting Phase 1 Development for Fuchs Endothelial Corneal Dystrophy (FECD) in 2024; Observational Study Currently Enrolling Patients
    CARLSBAD, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious degenerative genetic diseases, today announced first quarter 2024 financial results along with recent business highlights and upcoming milestones.
  • Design is on track to complete GLP studies for DT-216P2 by year-end 2024 to start patient trials in 2025.
  • R&D Expenses: Research and development (R&D) expenses were $9.8 million for the quarter ended March 31, 2024.
  • G&A Expenses: General and administrative (G&A) expenses were $4.6 million for the quarter ended March 31, 2024.

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

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화요일, 3월 19, 2024
Research, Observational study, Cerebellar ataxia, Biotechnology, Gene expression, FXN, Disease, Webcast, Patient, G&A, DM1, Design, IND, Treatment, Clinical trial, Investigational New Drug, Therapy, Completeness, Evaluation, DMPK, FA, HD, Food, FDA, GLP, HTT, Pharmaceutical industry

R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.

Key Points: 
  • R&D Expenses: Research and development (R&D) expenses were $11.0 million for the quarter ended December 31, 2023, and $57.1 million for the year ended December 31, 2023.
  • G&A Expenses: General and administrative (G&A) expenses were $4.1 million for the quarter ended December 31, 2023, and $21.1 million for the year ended December 31, 2023.
  • Net Loss: Net loss was $11.8 million for the quarter ended December 31, 2023, and $66.9 million for the year ended December 31, 2023.
  • Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $281.8 million as of December 31, 2023.

Dyne Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

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화요일, 3월 5, 2024
Therapy, Myotonia, Central nervous system, Research, DYN, Dystrophin, Safety, DMPK, DMD, Exercise, Standard of care, Conference, DM1, Eteplirsen, Muscular Dystrophy Association, Fatigue, Myotonic dystrophy, Patient, Clinical trial, PMO, Male, G&A, MDA, Episcopal conference, Pharmaceutical industry

WALTHAM, Mass., March 05, 2024 (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 fourth quarter and full year 2023 and recent 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 fourth quarter and full year 2023 and recent business highlights.
  • Net loss: Net loss for the quarter ended December 31, 2023 was $66.6 million, or $1.09 per basic and diluted share.
  • This compares with a net loss of $38.8 million, or $0.74 per basic and diluted share, for the quarter ended December 31, 2022.
  • Net loss for the year ended December 31, 2023 was $235.9 million, or $3.95 per basic and diluted share.

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy

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금요일, 3월 8, 2024
Pharmaceutical, Genetics, Health, Clinical Trials, Wheelchair, Myotonia, Cataract, DMPK, Wasting, Exercise, Arrowhead, Conference, DM1, RNA, Muscle weakness, Patient, CUG, Primate, MDA, RNA interference, Episcopal conference, Vaccine

Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.

Key Points: 
  • Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.
  • Pathogenesis of DM1 is driven by an expanded CUG trinucleotide repeat in the 3’-untranslated region of DMPK transcripts.
  • These abnormal transcripts cause mis-regulated splicing, known as spliceopathy, for certain messenger RNAs which are directly linked to the clinical manifestations of DM1.
  • Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

MBrace Therapeutics Appoints Steve Alley as Chief Scientific Officer

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화요일, 3월 5, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Genetics, Clinical Trials, Pfizer, Pathogenesis, Pennsylvania State University, DMPK, Cancer, Culture, Growth, Spacecraft, ADC, Linfield University, University, Therapy, Patient, MBrace, Chiron Corporation

MBrace™ Therapeutics, Inc. (“MBrace”), a clinical-stage biopharmaceutical company devoted to improving the lives of patients with cancer by developing novel antibody-drug conjugates (ADCs), today announced the appointment of Steve Alley, Ph.D., as chief scientific officer.

Key Points: 
  • MBrace™ Therapeutics, Inc. (“MBrace”), a clinical-stage biopharmaceutical company devoted to improving the lives of patients with cancer by developing novel antibody-drug conjugates (ADCs), today announced the appointment of Steve Alley, Ph.D., as chief scientific officer.
  • Dr. Alley joins MBrace with over 20 years of experience at Seagen, a world-leader in ADCs that was acquired by Pfizer in 2023.
  • “We are thrilled to welcome Dr. Alley to the MBrace team.
  • Dr. Alley has exceptional expertise in ADC development, and his vision will undoubtedly help propel the progress for our discovery and clinical programs,” said Isan Chen, M.D., president and chief executive officer at MBrace Therapeutics.

Ryvu Therapeutics to Present Preclinical Data on RVU120 and Synthetic Lethality Programs at the 2024 AACR Annual Meeting

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수요일, 3월 6, 2024
WBC, WRN, Lung cancer, RNA, Malignancy, Microsatellite instability, Proteome, Intellectual property, MMR, MPN, Glioblastoma, Neoplasm, Splenomegaly, DMPK, Phosphorylation, HDAC, STAT5, RNA-Seq, WES, Standard of care, Therapy, RecQ helicase, Phenotype, Ruxolitinib, DNA, DNA repair, Pancreatic serous cystadenoma, CDKN2A, Cancer, CRC, PIM, Arginine, MTA, Abstract, PDC, MTAP, Colorectal cancer, CRISPR, PV, MF, MSI-H, Cell, JAK, DNA mismatch repair, Medicine, Patient, Gene, PRMT5, Haematopoiesis, Mesothelioma, Pharmaceutical industry

Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.

Key Points: 
  • Ryvu's partner Menarini to present data on MEN1703 (SEL24), demonstrating promising anti-tumor activity in preclinical models of myelofibrosis both as a single agent and combined with ruxolitinib.
  • "We are excited to present our latest preclinical data at the AACR Annual Meeting, showcasing our significant progress in advancing novel small molecule therapies for oncology.
  • This year, we will present data from our most advanced preclinical project on MTA-cooperative PRMT5 inhibitors, the lead program within Ryvu's synthetic lethality pipeline.
  • Ryvu has developed potentially best-in-class MTA-cooperative PRMT5 inhibitors showing favorable drug-like properties and effective PRMT5 inhibition dependent on MTA binding.

ARTHEx Biotech Receives IND Clearance from FDA to Initiate the Phase I-IIa ArthemiR™ Trial of ATX-01 for Myotonic Dystrophy Type 1 (DM1)

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수요일, 2월 28, 2024
Research, MicroRNA, Messenger, FDA, Quality of life, DMPK, Food, Virginia Commonwealth University, DM1, Safety, Biomarker, MBNL, Muscle weakness, Myotonic dystrophy, Patient, Disease, Phase, Pharmaceutical industry

VALENCIA, Spain, Feb. 28, 2024 /PRNewswire/ -- ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S. Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1). 

Key Points: 
  • The ArthemiR™ trial is a Phase I-IIa double-blind, placebo-controlled, dose escalation study expected to enroll participants with classic Myotonic Dystrophy Type 1 (DM1).
  • ARTHEx will also investigate target engagement at the muscle level through biomarkers, including MBNL levels and splicing index.
  • In addition, the clinical endpoints from the trial will include measures related to muscle function, patient-reported outcomes, and quality of life measures.
  • Patients are eager for new trials, and we are delighted to start enrolment in the coming months."

Systemic Bio to Present Innovative Human-relevant Datasets from h-VIOS™ Platform at SOT and AACR 2024 Annual Meetings

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목요일, 2월 8, 2024
Abstract, Society of Toxicology, DMPK, Therapy, 3D Systems, CYP, AACR, SOT, Liver injury, Tissue, Liver, Urea, Research, P450, Annual general meeting

HOUSTON, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Systemic Bio™ , a 3D Systems (NYSE:DDD) company, is excited to announce the acceptance of two abstracts for presentation at the prestigious Society of Toxicology (SOT) 2024 Annual Meeting (Salt Lake City, March 10-14, 2024) and American Association for Cancer Research (AACR) 2024 Annual Meeting (San Diego, April 5-10, 2024).

Key Points: 
  • HOUSTON, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Systemic Bio™ , a 3D Systems (NYSE:DDD) company, is excited to announce the acceptance of two abstracts for presentation at the prestigious Society of Toxicology (SOT) 2024 Annual Meeting (Salt Lake City, March 10-14, 2024) and American Association for Cancer Research (AACR) 2024 Annual Meeting (San Diego, April 5-10, 2024).
  • At the SOT 2024 meeting, Systemic Bio will present its latest research on an advanced liver model that demonstrates several-fold enhanced albumin production, CYP (cytochrome P450) activity, and urea production compared to traditional models.
  • The second abstract, accepted for AACR 2024, showcases a novel model that simultaneously assesses on-target anti-tumor therapeutic efficacy and off-target liver injury of therapeutics across different modalities.
  • To delve deeper into this research, all conference attendees and fellow researchers can visit booth #4452 to engage directly with Systemic Bio’s team and discover the full potential of the h-VIOS platform.

Sai Life Sciences augments DMPK capabilities to ace large-scale collaborations

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목요일, 2월 1, 2024
Met, Pharmacokinetics, Growth, Peptide, Absorption, Pharmacology, Immunohistochemistry, Safety, IDD, TK, Appetite, Drug discovery, Proteolysis targeting chimera, DMPK, Automation, ADME, Metabolite, Metabolism, Toxicology, Development, Biology

HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .

Key Points: 
  • HYDERABAD, India, Feb. 01, 2024 (GLOBE NEWSWIRE) -- To offer global big pharma clients cutting-edge, end-to-end drug metabolism and pharmacokinetics (DMPK) services, Sai Life Sciences, a global Contract Research, Development & Manufacturing Organization (CRO-CDMO) , has expanded its DMPK capabilities .
  • Making the announcement, Krishna Kanumuri, CEO & Managing Director, Sai Life Sciences, said, “The focus of innovator companies on diversifying their supply chains bodes well for Indian CROs-CDMOs like ours.
  • Sauri Gudlavalleti, Chief Operating Officer said, “The latest expansion of DMPK capabilities augments our integrated drug discovery (IDD) offerings.
  • Sai Life Sciences also has plans to invest in niche areas such as peptides, oligos and large molecules to strengthen its discovery capabilities.

DP Technology Announces Nomination of Development Candidate, a Potential Best-in-Class Kv1.3 Inhibitor for Immunological Diseases Including IBD and AD

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화요일, 1월 30, 2024
Patient, D. E. Shaw Research, Simulation, Atopic dermatitis, Membrane, UC, Lilly, DESRES, RNA, Robots in Disguise, PPI, IBD, AI, Microsoft Dynamics 365, Algorithm, DMPK, Eli Lilly and Company, Drug development, GPX4, Antibody, Prevalence, Intrinsically disordered proteins, Calcineurin, Eurosport 1, KCNA3, Internally displaced person, Conditional sentence, TH1, Drug discovery, Inflammatory bowel disease, Vaccine

The nomination of novel Kv1.3 inhibitor DPT0218 for the treatment of various immunological diseases including IBD and AD.

Key Points: 
  • The nomination of novel Kv1.3 inhibitor DPT0218 for the treatment of various immunological diseases including IBD and AD.
  • Inhibition of Kv1.3 therefore offers a potential opportunity to specifically impair inflammatory immune cell activity and proliferation, including in antigen-activated T cells.
  • Long-lived memory T-cell populations, particularly tissue resident subsets, may contribute to the chronicity of IBD and represent a potential target for therapy[4].
  • It has shown positive results in preclinical animal models of various autoimmune diseases such as IBD and AD.