Exon

Tessera Therapeutics Highlights Advancements Across its Gene Writing™ and Delivery Platforms Including Proof of Concept Data in Non-Human Primates

Retrieved on: 
Monday, January 9, 2023
Phenylketonuria, Genome, Cell, Gene, PST, Flagship Pioneering, Therapy, Tissue, Biochemistry, AAV, Biotechnology, Health, DNA, PKU, Mutation, LNP, Conference, Stem cell, Disease, Hepatectomy, Exon, CAR, NHP, Allele, RNA transfection, Liver, Reverse transcriptase, Genetic distance, Sickle cell disease, Vaccine, Tessera

“Our preclinical studies demonstrate that we can deliver our Gene Writers to the right location, in the liver and beyond, and make therapeutic alterations to the genome.

Key Points: 
  • “Our preclinical studies demonstrate that we can deliver our Gene Writers to the right location, in the liver and beyond, and make therapeutic alterations to the genome.
  • The Company’s proprietary non-viral lipid nanoparticles (LNPs) are designed to deliver Gene Writers™ to targeted tissues, potentially enabling the application of Gene Writing™ therapies in vivo.
  • DNA Gene Writers™ are based on recombinase or transposase element biochemistry and have been engineered to integrate a therapeutically relevant payload of choice.
  • Additional data demonstrate the ability to write longer sequences and entire genes with high efficiency and specificity.

Entrada Therapeutics Announces Clinical Candidate, ENTR-601-45, for the Potential Treatment of People Living with Duchenne Muscular Dystrophy who are Exon 45 Skipping Amenable

Retrieved on: 
Monday, January 9, 2023
IND, Muscular Dystrophy Association, Therapy, MDA, RNA transfection, Duchenne muscular dystrophy, EEV, Entrada, Conference, DMD, Exon, Patient, Episcopal conference, Investigational New Drug, PMO, Pharmaceutical industry, Duchenne

The Company plans to submit an Investigational New Drug (IND) application in the second half of 2024.

Key Points: 
  • The Company plans to submit an Investigational New Drug (IND) application in the second half of 2024.
  • “There is a profound unmet need for people living with Duchenne and the more than 1,300 people in the US and Europe who are exon 45 skipping amenable,” said Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics.
  • Entrada plans to present additional data in support of ENTR-601-45 at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in March 2023.
  • ENTR-601-45 is designed to address the underlying cause of Duchenne muscular dystrophy due to mutated or missing exons in the DMD gene.

Deciphera Pharmaceuticals Announces Results from ctDNA Analysis from INTRIGUE Phase 3 Clinical Study Demonstrating Substantial Clinical Benefit of QINLOCK® in Second-Line GIST Patients with Mutations in KIT Exon 11 and 17/18 Only

Retrieved on: 
Tuesday, January 3, 2023
Biotechnology, Health, Pharmaceutical, Clinical Trials, Oncology, Dana–Farber Cancer Institute, Cancer, Progression-free survival, Gastrointestinal stromal tumor, INSIGHT, Common, DNA, ORR, Neoplasm, Trial of the century, PFS, GIST, Patient, FDA, Circulating tumor DNA, ITT, Women's Flat Track Derby Association Division 2, KIT, Exon, Pharmaceutical industry, Animal

“We are extremely pleased by the exploratory analysis showing that QINLOCK, already the standard of care for fourth-line GIST patients, provided substantial clinical benefit to this subgroup of second-line patients compared to sunitinib.

Key Points: 
  • “We are extremely pleased by the exploratory analysis showing that QINLOCK, already the standard of care for fourth-line GIST patients, provided substantial clinical benefit to this subgroup of second-line patients compared to sunitinib.
  • Primary mutations in KIT were detected in exon 11 in 157 patients and in exon 9 in 36 patients.
  • Common resistance mutations in KIT were detected in exons 17/18 in 89 patients and in exons 13/14 in 81 patients.
  • In patients with a KIT exon 11 primary mutation, 52 patients had mutations in exon 17/18 only, 41 had mutations in exon 13/14 only, and 22 patients had mutations in both exon 13/14 and exon 17/18.

Foundation Medicine Receives FDA Approval for FoundationOne®Liquid CDx as a Companion Diagnostic for a Certain Group of Tyrosine Kinase Inhibitors for Treatment of Non-Small Cell Lung Cancer Patients

Retrieved on: 
Wednesday, December 21, 2022
FDA, Health, Oncology, Cancer, Foundation Medicine, Liquid, Food, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Oncology, DNA, Patient, Gefitinib, TKI, FDA, Large-cell lung carcinoma, Neoplasm, Exon, EGFR, Pharmaceutical industry, Medical device, NSCLC

“For NSCLC patients whose tumors have EGFR exon 19 deletions or exon 21 substitutions, this approval opens new access avenues for targeted treatment options,” says Mia Levy, M.D., Ph.D., chief medical officer at Foundation Medicine.

Key Points: 
  • “For NSCLC patients whose tumors have EGFR exon 19 deletions or exon 21 substitutions, this approval opens new access avenues for targeted treatment options,” says Mia Levy, M.D., Ph.D., chief medical officer at Foundation Medicine.
  • “Following three recent group companion diagnostic approvals for Foundation Medicine’s tissue-based test, FoundationOne®CDx, this first group approval for FoundationOne Liquid CDx builds upon the momentum for more efficient and innovative regulatory approaches to the companion diagnostic approval process.
  • The current therapies for which FoundationOne Liquid CDx is a companion diagnostic under the group approvals are Tarceva (erlotinib), Tagrisso (osimertinib) and Iressa (gefitinib).
  • Moving forward, FoundationOne Liquid CDx will automatically become a companion diagnostic for future TKIs within this group for NSCLC that are approved by the FDA.

Ascidian Therapeutics to Present at the 41st Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Wednesday, January 4, 2023
Gene editing, Patient, ATP, RNA, ABCA4, Disease, Conference, AAV, Gene, Stargardt disease, Multimedia, Therapy, Publication, Ophthalmology, Retinopathy, Exon, Vaccine

BOSTON, Jan. 4, 2023 /PRNewswire/ -- Ascidian Therapeutics , a biotechnology company focused on treating human diseases by rewriting RNA, announced today President and CEO Romesh Subramanian, Ph.D., will present at the 41st Annual J.P. Morgan Healthcare Conference on January 10, 2023.

Key Points: 
  • BOSTON, Jan. 4, 2023 /PRNewswire/ -- Ascidian Therapeutics , a biotechnology company focused on treating human diseases by rewriting RNA, announced today President and CEO Romesh Subramanian, Ph.D., will present at the 41st Annual J.P. Morgan Healthcare Conference on January 10, 2023.
  • The presentation materials will be available upon request via the Publications & Presentations section of the Ascidian website following the live session.
  • Ascidian Therapeutics edits exons at the RNA level.
  • Ascidian Therapeutics, an ATP company, is redefining the treatment of disease by rewriting RNA.

Lung Cancer Research Foundation and MET Crusaders Announce Research Grant Awards

Retrieved on: 
Tuesday, December 6, 2022
Adenocarcinoma, Diagnosis, Partnership, Research, Patient, Cancer, Exon, NSCLC, Lung cancer, Crusades, MET, Quality of life, Survival, Cell, Education, Hope, Mutation, Pharmaceutical industry

NEW YORK, Dec. 6, 2022 /PRNewswire/ -- The Lung Cancer Research Foundation (LCRF) and MET Crusaders are pleased to announce funding of three grants focused on MET alteration-specific research. Each project is awarded $150,000 for two years, with a total investment of $450,000 for this grant cycle.

Key Points: 
  • NEW YORK, Dec. 6, 2022 /PRNewswire/ -- The Lung Cancer Research Foundation (LCRF) and MET Crusaders are pleased to announce funding of three grants focused on MET alteration-specific research.
  • This partnership has allowed MET Crusaders to leverage LCRF's research funding expertise to extend grant awards for innovative projects.
  • The 2022 Research Grants supported by the MET Crusaders LCRF partnership include:
    The Lung Cancer Research Foundation (LCRF) is the leading nonprofit organization focused on funding innovative, high-reward research with the potential to extend survival and improve quality of life for people with lung cancer.
  • MET Crusaders is a trade name of the MET Cancer Foundation, a 501c3 charitable foundation.

Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy

Retrieved on: 
Monday, October 31, 2022
DMD, U.S. Securities and Exchange Commission, Marketing, Safety, PMO, Diaphragm, Time, Transferrin, Clinical trial, Accelerated approval (FDA), Food, Dystrophin, Priority review, Degenerative disease, Therapy, Muscle, FSHD, Private Securities Litigation Reform Act, Heart, Second Half, Patient, SEC, Terminology, DM1, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Mortality, Security (finance), FORCE, Duchenne muscular dystrophy, Disease, Company, Form 10-Q, DYN, GLOBE, Myopathy, Nasdaq, FDA, MPH, Exon, Plasma protein binding, COVID-19, Facioscapulohumeral muscular dystrophy, Review, MAD, Fast Track, Cardiac muscle, Pharmacokinetics, Pharmaceutical industry, Dyne, Facebook

WALTHAM, Mass., Oct. 31, 2022 (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 announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping. DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial.

Key Points: 
  • A drug receiving Fast Track designation also may be eligible for Accelerated Approval and Priority Review if relevant criteria are met.
  • DYNE-251 is Dynes product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
  • Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases.
  • Dyne has a broad portfolio of programs for serious muscle diseases, including candidates for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).

Theseus Pharmaceuticals Presents Preclinical Data Characterizing THE-349, its Fourth-Generation EGFR Inhibitor in Development for NSCLC, at the 34th EORTC-NCI-AACR Symposium

Retrieved on: 
Wednesday, October 26, 2022
U.S. Securities and Exchange Commission, SEC, PD, IND, DTC, Non-small-cell lung carcinoma, NASDAQ, ENA, Research and development, Patient, Neoplasm, Security (finance), PDX, KIT, GIST, Cancer, D, Private Securities Litigation Reform Act, Lung cancer, Company, LTC, NSCLC, TKI, CNS, Recurrence, Survival, Brain, Forward-looking statement, Theseus, LC, Exon, Risk, Large-cell lung carcinoma, Pharmaceutical industry, Vaccine, Medical imaging, EGFR, Research, T790M

CAMBRIDGE, Mass., Oct. 26, 2022 /PRNewswire/ -- Theseus Pharmaceuticals, Inc. (NASDAQ: THRX) (Theseus or the Company), a clinical-stage biopharmaceutical company focused on improving the lives of cancer patients through the discovery, development, and commercialization of transformative targeted therapies, today announced preclinical data characterizing its fourth-generation epidermal growth factor receptor (EGFR) inhibitor, THE-349, will be detailed in a live poster presentation at the 34th EORTC-NCI-AACR Symposium on molecular targets and cancer therapeutics (ENA), taking place in Barcelona, Spain on October 26 – 28, 2022. THE-349 is currently in preclinical development as a single-molecule inhibitor of all major classes of EGFR activating and resistance mutations consisting of single-, double- and triple-mutant EGFR variants, including T790M and C797X, for treatment of EGFR-mutant non-small cell lung cancer (NSCLC).

Key Points: 
  • "We are pleased to share preclinical data characterizing THE-349, our newly nominated fourth-generation EGFR inhibitor," said William Shakespeare, Ph.D., President of Research and Development at Theseus.
  • These preclinical data demonstrate that THE-349, as a single agent, exhibits highly potent inhibition of all major EGFR mutant variants with a high degree of kinome and wild-type EGFR selectivity.
  • The activity profile achieved by THE-349 positions the asset for development as a single agent and potentially in combination with other, non-EGFR inhibitor modalities.
  • Deep and/or complete tumor regression in an osimertinib-resistant, patient-derived (PDX) NSCLC model harboring heterogeneous EGFR mutant variants (D and DTC).

Dyne Therapeutics Presents Preclinical Data from its Duchenne Muscular Dystrophy Programs Targeting Exons 51 and 53 at World Muscle Society 2022 Congress

Retrieved on: 
Tuesday, October 11, 2022
LinkedIn, Securities and Exchange Commission (Philippines), Muscle, Time, Congress, FSHD, Form 10-Q, Therapy, Disease, SEC, World Muscle Power Classic, MAD, Private Securities Litigation Reform Act, Poster, Degenerative disease, Facioscapulohumeral muscular dystrophy, Twitter, Security (finance), DM1, Mortality, DYN, Clinical trial, Diaphragm, Nasdaq, FORCE, DMD, Duchenne muscular dystrophy, Mutation, Cardiac muscle, Transferrin, Pharmacokinetics, Dystrophin, Safety, GLOBE, Heart, GLP, Myopathy, Terminology, COVID-19, Exon, Toxicology, PMO, Pharmaceutical industry, Vaccine, Dyne, Facebook, Patient

WALTHAM, Mass., Oct. 11, 2022 (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 announced the presentation of preclinical data from its Duchenne muscular dystrophy (DMD) programs for the treatment of individuals with DMD mutations amenable to skipping exons 51 or 53. The data are featured in a poster at the 27th International Hybrid Annual Congress of the World Muscle Society taking place in Halifax, Nova Scotia, Canada from October 11-15, 2022.

Key Points: 
  • The data are featured in a poster at the 27th International Hybrid Annual Congress of the World Muscle Society taking place in Halifax, Nova Scotia, Canada from October 11-15, 2022.
  • The poster being presented at World Muscle features foundational preclinical data underpinning the clinical development program for DYNE-251 and the first in vitro data for a FORCE conjugate targeting exon 53, said Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne.
  • Dynes FORCE platform targets the transferrin receptor 1, which is highly expressed on the surface of muscle cells.
  • DYNE-251 is Dynes product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Precipio Signs Agreement with Another Major US Healthcare Distributor for HemeScreen™

Retrieved on: 
Thursday, September 15, 2022
Securities Exchange Act of 1934, Laboratory, Efficiency, COVID-19, Policy, Securities Act of 1933, Gene, Growth, Government, Patient, U.S. Securities and Exchange Commission, Exon, Annual report, Coronavirus, NASDAQ, Pols, GLOBE, Risk management, HAVEN

Precipio continues to execute its HemeScreen distribution growth strategy of a multi-pronged approach targeting national and regional reference labs, hospital networks and physician-owned laboratories (POLs).

Key Points: 
  • Precipio continues to execute its HemeScreen distribution growth strategy of a multi-pronged approach targeting national and regional reference labs, hospital networks and physician-owned laboratories (POLs).
  • The diversity of distributors and channel partners engaging with Precipio maximizes coverage of the full market potential.
  • The new distributor focuses on a segment of the healthcare business which, by our estimate, represents another $100M of market potential that our current distributors are less likely to reach.
  • With this distributor, we now have >250 sales representatives in the field calling on various groups of qualified potential customers.