Exon

Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 6, 2024
Therapy, Facial muscles, Pharmacology, Central nervous system, Research, GSK, MD, Liver disease, Dystrophin, Obesity, Cerebrospinal fluid, N-Acetylgalactosamine, HD, Weight loss, DMD, Duchenne muscular dystrophy, Lof, Exercise, Lipolysis, Exon, MBA, Conference, Wave, Activin and inhibin, AATD, Health, Safety, Biomarker, RNA, Greenshoe, Genetics, Pharmacokinetics, Webcast, Patient, Clinical trial, HTT, Q&A, Small RNA, Serum, Episcopal conference, Human, Coronary artery disease, Pharmaceutical industry

In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.

Key Points: 
  • In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.
  • Revenue was $29.1 million for the fourth quarter of 2023 as compared to $1.2 million in the prior year quarter.
  • Net loss was $16.3 million for the fourth quarter of 2023 as compared to $43.7 million in the prior year quarter.
  • ET to review the fourth quarter and full year 2023 financial results and pipeline updates.

Wave Life Sciences Announces Upcoming Presentations at MDA Conference that Highlight Best-in-Class Potential for WVE-N531 in Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024
B cell, Therapy, Pharmacology, Diaphragm, Chemistry, Dystrophin, DMD, Part, Facial muscles, Duchenne muscular dystrophy, Exon, Conference, Wave, Health, Muscular Dystrophy Association, RNA, PN, Optimism, Protein, Oligonucleotide, RAPS, Half-life, MDA, Episcopal conference, Dietary supplement

CAMBRIDGE, Mass., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced its upcoming presentations at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 3-6 in Orlando, FL.

Key Points: 
  • Wave’s poster presentations will highlight the best-in-class potential of WVE-N531 in Duchenne muscular dystrophy (DMD), which is currently being evaluated in the Phase 2, potentially registrational FORWARD-53 study.
  • The presentations will also illustrate the impact of Wave’s novel PN chemistry on pharmacology of its exon skipping oligonucleotides.
  • These data suggest that WVE-N531 muscle concentrations in the clinic may be higher in heart and diaphragm than in skeletal muscle.
  • “At Wave, we increasingly continue to regard exon skipping as the preferred mechanism for altering DMD disease progression in those amenable to this approach.

Twist Bioscience Launches IVDR-compliant Precision Dx Products for Whole Exome Sequencing

Retrieved on: 
Tuesday, March 5, 2024
Research, Other Health, Pharmaceutical, Technology, Medical Devices, Genetics, Health Technology, Science, Nanotechnology, Biotechnology, Medical Supplies, Health, Other Science, Twist Bioscience, Twist, Reagent, Silicon, IVDR, NGS, Documentation, Doctor of Philosophy, Safety, Certification, Exon, Genome, Laboratory, DNA, IVD, Workflow, Medical device

The suite of our CE-marked Products includes:

Key Points: 
  • The suite of our CE-marked Products includes:
    The Twist Precision Prep and Enrichment Dx Kit1 (Reagents for enzymatic fragmentation, library preparation, and target enrichment), which is panel-agnostic2
    Twist Precision Exome Dx Kit1 (Reagents for enzymatic fragmentation, library preparation, and target enrichment along with targeted capture probes for exome enrichment; IVDR compliant workflow)
    The Twist Precision Prep and Enrichment Dx Kit and the Twist Precision Exome Dx Panel can be used individually within existing workflows or as a combined solution known as the Twist Precision Exome Dx Kit.
  • “The Twist Precision Dx products are our first NGS product line specifically developed to be included within regulated customer products.
  • The Twist Precision Exome Dx Panel1 is a qualified Twist DNA panel that can be used in conjunction with the Twist Precision Prep and Enrichment Dx kit to produce exome-enriched libraries compatible for NGS applications.
  • Twist Precision Exome Dx Kit1 utilizes the qualified Twist Precision Exome Dx Panel along with the Twist Precision Prep and Enrichment Dx Kit to produce exome-enriched libraries compatible for NGS applications.

Whole Genome Sequencing Market Global Industry Forecast Report 2018-2028, Featuring Profiles of Illumina, Agilent Technologies, 10 x Genomics, BGI, Oxford Nanopore, Nebula Genomics & Veritas Genetics

Retrieved on: 
Wednesday, January 31, 2024
Cloud, Volcanic explosivity index, Whole genome sequencing, Growth, Microbiology, Workflow, WES, Genomics, Research, AI, DNA, Bacteria, Government, Gene, Mutation, Diagnosis, Microorganism, Exon, Competition, Exome sequencing, Sequence, Next Generation, NGS, Cloud computing, Cancer, Genome, Infection, Antibiotics

DUBLIN, Jan. 31, 2024 /PRNewswire/ -- The "Whole Genome Sequencing Market - Global Industry Size, Share, Trends, Competition, Opportunity, and Forecast, 2018-2028" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • DUBLIN, Jan. 31, 2024 /PRNewswire/ -- The "Whole Genome Sequencing Market - Global Industry Size, Share, Trends, Competition, Opportunity, and Forecast, 2018-2028" report has been added to ResearchAndMarkets.com's offering.
  • The Whole Genome Sequencing Market is expected to grow with an impressive CAGR in the forecast period 2024-2028.
  • Increasing technological advancement in NGS instruments and update in technology utilized in Sequencing techniques are adding to further drive the sequencing market growth in the forecast period.
  • In this report, Whole Genome Sequencing Market has been segmented into the following categories:

Deciphera Pharmaceuticals Announces Nature Medicine Publication of Results from Exploratory ctDNA Analysis from INTRIGUE Phase 3 Study Demonstrating Substantial Clinical Benefit of QINLOCK® in 2L GIST Patients with Mutations in KIT Exon 11 and 17/18

Retrieved on: 
Friday, January 5, 2024
Research, Clinical Trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Oncology, Other Science, Patient, Gastrointestinal stromal tumor, Survival, Risk, Neoplasm, INSIGHT, Progression-free survival, Disease, KIT, DNA, Cancer, Liquid biopsy, Exon, Death, Nature Medicine, Manuscript, Overalls, PFS, Sunitinib, AP, GIST, Pharmaceutical industry

“The results published in Nature Medicine provide compelling evidence that QINLOCK may provide progression-free and overall survival benefit to second-line (2L) GIST patients in whom a liquid biopsy reveals primary KIT exon 11 mutations plus secondary mutations restricted to KIT exons 17 and 18.

Key Points: 
  • “The results published in Nature Medicine provide compelling evidence that QINLOCK may provide progression-free and overall survival benefit to second-line (2L) GIST patients in whom a liquid biopsy reveals primary KIT exon 11 mutations plus secondary mutations restricted to KIT exons 17 and 18.
  • “Our ongoing INSIGHT pivotal Phase 3 study is designed to confirm the exceptional efficacy we observed in this exploratory analysis from INTRIGUE.
  • A prespecified exploratory objective in INTRIGUE was to evaluate anti-tumor efficacy of QINLOCK according to baseline KIT primary and secondary mutation status.
  • In patients with a detectable KIT exon 11 primary mutation (n=157), 52 patients also had mutations in KIT exon 17/18 only and 41 had mutations in KIT exon 13/14 only.

Capricor Therapeutics Reports Third Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Tuesday, November 14, 2023
Trigger, Annual general meeting, Chemistry, Patient, Small RNA, Exosome, PLOS, Kidney, Webcast, Therapy, Exon, Business, MUSCULAR, Employment, Food, International, PUL, LVEF, RRM2, Ovarian cancer, Duchenne muscular dystrophy, MRI, Horizon, Commercialization, Amgen, WMS, ID, AAEV, BLA, Cohort, Vaccine, Tissue, Distribution deal, PPMD, Asos, Partnership, Biologics license application, DMD, Apoptosis, Distribution, Liver, Protein, Biotechnology, Cohort study, Mouse, Poster, Omicron, FDA, Seminal vesicles, File, CMC, HIV disease progression rates, Dietary supplement, Mobile phone, Pharmaceutical industry, Fine chemical, Video game, Nursing

SAN DIEGO, Nov. 14, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, announced today its financial results for the third quarter ended September 30, 2023 and provided a corporate update.

Key Points: 
  • Next steps for Cohort A:
    Plan to report the outcome from the interim futility analysis in the fourth quarter of 2023.
  • Held a positive Type-B clinical meeting with FDA in the third quarter of 2023.
  • Next steps for Cohort B:
    Expect to commence enrollment for Cohort B in the fourth quarter of 2023.
  • Revenues for the third quarter of 2023 were approximately $6.2 million compared with approximately $1.6 million for the third quarter of 2022.

Gene Splicing Reduces Effectiveness of CD20-Targeting Monoclonal Antibodies Designed to Treat Variety of Blood Cancers and Disorders

Retrieved on: 
Friday, November 17, 2023
DOI, DRS, Malignancy, CHOP, Research, University, Bone marrow, Leukemia, Blood, Hospital, Protein, Jim Valvano, Patient, Lymphoma, Division, Follicular lymphoma, Messenger, Child, Medical laboratory, Virus, Burkitt lymphoma, Antibody, Exon, Children's Hospital of Philadelphia, Doctor of Philosophy, Emerson Collective, MS4A1, Alternative splicing, Bangladesh Technical Education Board, Cell, RNA, Vaccine, Pathology, CD20

PHILADELPHIA, Nov. 17, 2023 /PRNewswire/ -- Immunotherapies that target the CD20 antigen have revolutionized how patients with a variety of blood cancers and hematologic disorders have been treated. However, many patients develop resistance to these treatments due to a loss of the antigen that's being targeted. Now, a new study from researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania (Penn) has found that gene splicing occurring within these cells can cause significant changes in CD20 protein levels that render the therapies ineffective.

Key Points: 
  • PHILADELPHIA, Nov. 17, 2023 /PRNewswire/ -- Immunotherapies that target the CD20 antigen have revolutionized how patients with a variety of blood cancers and hematologic disorders have been treated.
  • These findings may lead to more appropriate choices for therapy that maximize benefits to patients affected by a variety of blood cancers.
  • CD20 is a cell-surface protein involved in the fine-tuning of B cell responses to foreign agents like viruses.
  • Prior research had shown that a loss of CD20 reduced the effectiveness of these immunotherapies, since it would remove their intended target.

Wave Life Sciences Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 9, 2023
Research, Q&A, Dystrophin, DSM-IV codes, Biomarker, Therapy, Obesity, CSF, RNA, Pizzicato, Human, HD, MD, Coronary artery disease, Liver, CNS, Exon, MBA, Liver disease, Oligonucleotide, Pharmacokinetics, CTAS, Clinical trial, Webcast, Messenger, GSK, Serum, AATD, Safety, DMD, N-Acetylgalactosamine, Patient, Small RNA, NHP, Mutation, Vaccine, Medical imaging, Cryptocurrency, Pharmaceutical industry, Takeda, Wave

ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the third quarter ended September 30, 2023, and provided a business update.
  • Wave expects to initiate dosing with WVE-006 in healthy volunteers in the fourth quarter of 2023 and deliver proof-of-mechanism data in individuals with AATD in 2024.
  • Wave remains on track to initiate dosing in FORWARD-53 in 2023 and enroll up to 10 boys in the trial.
  • ET to review third quarter 2023 financial results and pipeline updates.

Ascidian Therapeutics Raises $40 Million in Series A Extension Financing from Apple Tree Partners and Appoints Michael Ehlers as Interim CEO

Retrieved on: 
Wednesday, November 8, 2023
FDA, IND, AAV, Trans-splicing, Food, Therapy, ATP, RNA, ABCA4, Retinopathy, Ascidian mitochondrial code, Exon, Regulation of tobacco by the U.S. Food and Drug Administration, Society, Cell, Stargardt disease, Risk, Gene, Animal, Drug discovery, DNA, Pharmaceutical industry, Vaccine, Stargardt, Ascidiacea

BOSTON, Nov. 8, 2023 /PRNewswire/ -- Ascidian Therapeutics, a biotechnology company focused on treating human diseases by rewriting RNA, today announced $40 million in Series A extension funding committed by Apple Tree Partners (ATP). The funds will be used to advance Ascidian's lead program, an RNA exon editor to halt the progression of Stargardt disease or other ABCA4 retinopathies, and additional programs in the company's pipeline. The company also announced that its founding CEO Michael Ehlers, M.D., Ph.D., will return as interim President and Chief Executive Officer, effective immediately. Dr. Ehlers, who also chairs Ascidian's Board of Directors and serves as Chief Scientific Officer at ATP as well as a venture partner at the firm, succeeds Romesh Subramanian, Ph.D., who is departing Ascidian.

Key Points: 
  • The company also announced that its founding CEO Michael Ehlers, M.D., Ph.D., will return as interim President and Chief Executive Officer, effective immediately.
  • Dr. Ehlers, who also chairs Ascidian's Board of Directors and serves as Chief Scientific Officer at ATP as well as a venture partner at the firm, succeeds Romesh Subramanian, Ph.D., who is departing Ascidian.
  • Today's announced financing follows ATP's initial $50 million Series A investment in Ascidian announced in October 2022.
  • Beyond its lead program, Ascidian is advancing programs with first-in-class potential in neurological and neuromuscular disorders.

IDRx Presents Preliminary Clinical Data from Ongoing Phase 1 StrateGIST Study at CTOS 2023 Supporting Best-in-Class Potential of IDRX-42 in Patients with GIST

Retrieved on: 
Thursday, November 2, 2023
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, TKI, Patient, KIT, PRS, MTD, MPH, Physician, BID, Exon, Poster, Strategist, UZ Leuven, Annual general meeting, GIST, RECIST, Stromal cell, Therapeutic index, Pharmaceutical industry, MD

“Importantly, these preliminary data will help guide our discussions with physicians regarding development strategies for IDRX-42 in patients with GIST in early lines of therapy to prevent the emergence of genomically-driven resistance mutations.

Key Points: 
  • “Importantly, these preliminary data will help guide our discussions with physicians regarding development strategies for IDRX-42 in patients with GIST in early lines of therapy to prevent the emergence of genomically-driven resistance mutations.
  • Median duration on treatment was 16 weeks and continuing, with 70% (23/33) of patients remaining on treatment at time of data cutoff.
  • 4/28 patients are on study for
  • Title: Phase 1 Study of IDRX-42 in Patients with Advanced Gastrointestinal Stromal Tumors Resistant to Prior Systemic Therapy: Early Results
    Session: CTOS Poster Reception 5:30-6:30 p.m. GMT, Thursday, November 2nd