DMPK

NeuBase Presents New Preclinical Data at ASGCT 2022 for Its DM1 Program Demonstrating Wide Tissue Distribution and Supporting a Differentiated Whole-Body Treatment Solution

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화요일, 5월 17, 2022
Company, Disease, DM1, Medicine, Annual general meeting, Private Securities Litigation Reform Act, RNA splicing, U.S. Securities and Exchange Commission, Nasdaq, Myotonia, Patient, Therapy, Society, DMPK, Marketing, Research, Gene, CNS, Government budget balance, Intention, Plasma, Dietrich Stephan, Biotechnology, RNA, Muscle tissue, Muscle weakness, Risk, American Society of Gene and Cell Therapy, Mutation, Cell, PK, PNA, Tissue, GLOBE, Pharmaceutical industry, Biodistribution, COVID-19

NT-0231.F rapidly cleared the plasma, and each tissue evaluated displayed an extended elimination phase with tissue concentrations measurable for at least four weeks following a single IV dose administration.

Key Points: 
  • NT-0231.F rapidly cleared the plasma, and each tissue evaluated displayed an extended elimination phase with tissue concentrations measurable for at least four weeks following a single IV dose administration.
  • Sandra Rojas-Caro, M.D., Chief Medical Officer of NeuBase, said, We continue to build a robust and compelling data set potentially supporting a whole body treatment solution for DM1.
  • These PK and biodistribution data presented today show IV administration of our lead candidate results in exposure in the major tissues that are affected by DM1.
  • We have now demonstrated in preclinical models that our lead candidate NT-0231.F achieves clinically relevant molecular and functional rescue as well as whole-body distribution.

Dyne Therapeutics Presents New In Vivo Data from DYNE-101 at ASGCT Annual Meeting Demonstrating Low Monthly Dosing Leads to Robust DMPK RNA Knockdown

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월요일, 5월 16, 2022
People, Myopathy, Phenotype, RNA transfection, Disease, DM1, Annual general meeting, Toxicology, U.S. Securities and Exchange Commission, Nasdaq, ASO, Myotonia, Patient, Duchenne muscular dystrophy, GLP, Facioscapulohumeral muscular dystrophy, Private Securities Litigation Reform Act, Therapy, DYN, Muscle, Digestion, Clinical trial, Degenerative disease, DMD, Society, DMPK, COVID-19, Form 10-Q, Kidney, Gene, IND, Cardiac muscle, Lists of diseases, Cognition, Toxicity, FORCE, Transferrin, RNA, Terminology, Safety, FSHD, LinkedIn, Western literature, Twitter, Cell, SEC, Dyne, Security (finance), Letter, CTG, Speech, GLOBE, Vaccine, Pharmaceutical industry, Facebook

WALTHAM, Mass., May 16, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, is presenting new in vivo data from DYNE-101, its myotonic dystrophy type 1 (DM1) candidate, at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting today demonstrating robust knockdown of DMPK RNA in multiple muscles with low monthly dosing.

Key Points: 
  • These findings combined with previous data demonstrating correction of splicing, support the potential for DYNE-101 to be a disease-modifying therapy with low, infrequent dosing.
  • We look forward to evaluating DYNE-101 in our planned global multiple ascending dose clinical trial in patients with DM1.
  • As a result of this altered splicing, people living with DM1 typically experience progressive weakness of skeletal, cardiac and smooth muscle.
  • The new data being presented during ASGCT evaluated monthly repeat doses of DYNE-101 in an innovative hTfR1/DMSXL mouse model developed by Dyne and in non-human primates (NHPs).

Sygnature Discovery to Deploy Iktos’s AI for Drug Design Software Makya™

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화요일, 5월 10, 2022
Research, Technology, Other Technology, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Cloud, Drug design, Deep learning, II, Safety, Software, CA, Drug discovery, International trade, Doctor of Philosophy, DMPK, Pharmacology, Czech Game of the Year Awards, III, Analysis, QSAR, Therapy, CRO, Artificial intelligence, De novo, Project Jupyter, VPC, College of Arts and Sciences, Efficiency, Disease, Virtual private cloud, Intelligence, Enterprise, Software as a service, Solution, Technology, CEO, AI, MPO, Medical device, Pharmaceutical industry, Ikto!: 20 Megales Epitihies Tou

Iktos, a company specialized in Artificial Intelligence (AI) for new drug design and Sygnature Discovery, a leading integrated drug discovery CRO headquartered in Nottingham, UK, with expertise across a range of therapeutic and biological target classes today announced a collaboration agreement in AI for new drug design.

Key Points: 
  • Iktos, a company specialized in Artificial Intelligence (AI) for new drug design and Sygnature Discovery, a leading integrated drug discovery CRO headquartered in Nottingham, UK, with expertise across a range of therapeutic and biological target classes today announced a collaboration agreement in AI for new drug design.
  • Under the 3-year agreement, Sygnature will deploy Iktos de novo generative design software Makya, which will be used by Sygnature scientists to facilitate rapid and efficient design of novel compounds and accelerate hit-to-lead/lead optimisation, reinforcing Sygnature Discoverys drug design expertise delivering its integrated drug discovery solutions to its world-wide customer base.
  • In the recent years, Iktos has emerged as one of the world leaders in AI for drug design, establishing multiple collaborations with renowned pharmaceutical companies and successfully developing the AI software platforms Makya for new drug design and Spaya for synthesis planning.
  • Sygnature Discovery serves its growing customer base with fully integrated drug discovery services from target validation through to preclinical candidate selection.

NeuBase Therapeutics Announces Presentations at the American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting

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월요일, 5월 2, 2022
Calendar, PNA, Mutation, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, RNA, Company, Medicine, Government budget balance, Food, Patient, DMPK, Risk, DM1, Regulation of tobacco by the U.S. Food and Drug Administration, Marketing, Intention, Investigational New Drug, RNA splicing, SEC, Technology, Research, Annual general meeting, Vaccine, COVID-19

The PNA pharmacophore is conjugated to NeuBases novel delivery technology that is designed for broad distribution, including into the deep brain, with the potential for a whole body, disease-modifying solution for DM1.

Key Points: 
  • The PNA pharmacophore is conjugated to NeuBases novel delivery technology that is designed for broad distribution, including into the deep brain, with the potential for a whole body, disease-modifying solution for DM1.
  • NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines that Drug the Genome.
  • This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act.
  • Our forward-looking statements contained herein speak only as of the date of this press release.

Dyne Therapeutics Reports First Quarter 2022 Financial Results and Business Highlights

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월요일, 5월 2, 2022
Well, COVID-19, U.S. Securities and Exchange Commission, Form 10-Q, Myopathy, IND, Nasdaq, Twitter, DYN, GLOBE, Facioscapulohumeral muscular dystrophy, Company, Private Securities Litigation Reform Act, Cell, Food, Research, Degenerative disease, Patient, MAD, Security (finance), Gene, DMPK, Clinical trial, FDA, Letter, DM1, Pathology, Therapy, Duchenne muscular dystrophy, FORCE, R, LinkedIn, Society, Investigational New Drug, FSHD, SEC, DMD, G&A, Terminology, Pharmaceutical industry, Dyne, Facebook

WALTHAM, Mass., May 02, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a 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 2022 and business highlights.

Key Points: 
  • WALTHAM, Mass., May 02, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a 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 2022 and business highlights.
  • Dyne expects to submit an IND for DYNE-301 in facioscapulohumeral muscular dystrophy (FSHD) in the second half of 2022.
  • Research and development (R&D) expenses: R&D expenses were $28.2 million for the quarter ended March 31, 2022, compared to $18.6 million for the quarter ended March 31, 2021.
  • Net loss: Net loss for the quarter ended March 31, 2022 was $35.6 million, or $0.69 per basic and diluted share.

Awakn Successfully Completes Phase One of Its Drug Discovery Program

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수요일, 4월 6, 2022
Company, Special-purpose acquisition company, FSE, Human, Therapy, Forward-looking statement, Pharmacology, SEDAR, Addiction, Public opinion, NCE, Perception, Person, NEO, DMPK, Industry, Review, Intention, Goal, Time, Phrase, News, MDMA, Acquisition, Pharmaceutical industry, Audit, Security (finance)

Two Chemical Series Identified to Facilitate Lead Optimisation Activities

Key Points: 
  • Awakn has completed a hit to lead program which delivered its key goals of identifying and patenting novel chemistry scaffolds.
  • It also established drug discovery assays with the potential to facilitate lead optimization activities.
  • Professor David Nutt, Awakn's Chief Research Officer commented, "I am so pleased that this unique and innovative program has progressed so quickly.
  • Awakn is building a strong and unique portfolio of targeted novel candidates to treat both substance and behavioural addictions.

GHIT Fund Announces New Investments: A Total of 740 Million Yen for Malaria Drugs and Vaccine

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목요일, 3월 31, 2022
Malaria, Government of Japan, World Health Organization, WHO, UNDP, Investment, Volunteering, Bill & Melinda Gates Foundation, Continuity, Wellcome, Vaccine, Bill, Research, World, PPP, DMPK, CS, Parasitism, United Nations Development Programme, Safety, Neutralizing antibody, Plasmodium vivax, United Nations, US, Plasmodium falciparum, Tuberculosis, RTS, Patient, Partnership, Immunization, Pharmaceutical industry

Existing innovations alone are not sufficient to combat malaria," said GHIT CEO Osamu Kunii.

Key Points: 
  • Existing innovations alone are not sufficient to combat malaria," said GHIT CEO Osamu Kunii.
  • We hope that these new innovations will be important tools in the malaria elimination strategy and bring greater impact for patients."
  • Preclinical development of a monoclonal antibody to prevent P. falciparum malaria
    GHIT will invest 539 million yen (US$4.7 million) for the development of monoclonal antibodies (mAbs) for malaria prevention..
  • The GHIT Fund mobilizes the Japanese industry, academia, and research institutes to create new drugs, vaccines, and diagnostics for malaria, tuberculosis, and neglected tropicaldiseases, in collaboration with global partners.

CLD Partners Announced Launch of Professional Services Quoting Application, PSQuote

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금요일, 3월 25, 2022
Business development, Organization, Growth, Multimedia, Pharmacokinetics, Cros, FinancialForce.com, PSA, DMPK, Contract, CRM, CRO, Salesforce, CLD, Professional services automation, Contact, Fine chemical, Public relations

PSQuote is a newly developed professional services quoting application built on the Salesforce platform (PSQuote is a wholly-owned division of CLD Partners).

Key Points: 
  • PSQuote is a newly developed professional services quoting application built on the Salesforce platform (PSQuote is a wholly-owned division of CLD Partners).
  • CLD Partners has engaged with AlliancePharma since June 2021, for FinancialForce implementation services, and this new deployment is the next step in a growing business relationship.
  • Over the years, CLD Partners has built many custom professional services estimation tools based on the Salesforce platform, including for many large enterprises.
  • CLD Partners CEO, Steve Clune, who originally designed PSQuote, sees this as a further step in CLD offering specific expertise to the CRO space.

HitGen Announces Research Collaboration Focused on DNA-Encoded Library Based Drug Discovery

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목요일, 3월 17, 2022
Howard Hughes Medical Institute, FFS, Organization, Duke University, DNA, Biochemistry, HitGen Inc., Shanghai Stock Exchange, GPCR, Principal investigator, PK, Multimedia, Board, HHMI, Pharmacology, IND, Duke University Hospital, Research, CMC, Program, PD, Macrocycle, DMPK, Nobel Prize in Chemistry, Computational chemistry, Cell biology, Nobel Prize, Technology, Industry, Acquisition, Foundation, Cardiology, Drug discovery, Agrochemical, Dels, Efficiency, DEL, Duke University Medical Center Patient Rapid Transit, Biophysics, File, Pharmaceutical industry, Fine chemical, Medicine, Chemistry

OpenDEL is an affordable self-service drug discovery DEL platform for start-ups, biotechs, and academic researchers.

Key Points: 
  • OpenDEL is an affordable self-service drug discovery DEL platform for start-ups, biotechs, and academic researchers.
  • HitGen's full range of DEL products and the efficiency of the screening process have made it possible for HitGen to enable drug discovery projects for many organizations around the world.
  • HitGen became a publicly listed company in Shanghai Stock Exchange in April 2020 (ticker code 688222.SH) and has established a drug discovery research platform for small molecules and nucleic acid drug centered on the design, synthesis and screening of DNA encoded chemical libraries (DELs), fragment-based drug discovery (FBDD) and structure-based drug design (SBDD) technologies.
  • HitGen has approximately 20 in-house drug discovery programs at different stages of research & development.

NeuBase Therapeutics Presents New Preclinical Data at MDA 2022 for Its Myotonic Dystrophy Type 1 Program Demonstrating Splice Rescue, Nuclear Aggregate Resolution, and Myotonia Reversal

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일요일, 3월 13, 2022
Nasdaq, EDT, RNA splicing, Patient, Private Securities Litigation Reform Act, Congress, Company, Marketing, Research, IND, Webcast, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Wasting, Government budget balance, Tissue, Mutation, Investor, U.S. Securities and Exchange Commission, Conference, Risk, Investigational New Drug, DM1, Muscle weakness, Intention, GLOBE, PNA, Calendar, Technology, Dietrich Stephan, Episcopal conference, Disease, Therapy, Smart contract, SEC, Food, Poster, Biotechnology, RNA, Medicine, Drug discovery, DMPK, Pharmaceutical industry, Medical imaging, Vaccine, Myotonia, COVID-19

We are excited to present new preclinical data for our lead candidate, NT-0231.F, to support our differentiated approach for DM1.

Key Points: 
  • We are excited to present new preclinical data for our lead candidate, NT-0231.F, to support our differentiated approach for DM1.
  • Initial pharmacokinetic data supports a whole-body solution to the disease, and additional preclinical work is in progress to assess biodistribution in key tissues.
  • These data are an important milestone in our DM1 development program and support our pursuit of a best-in-class therapeutic profile for the disease.
  • A single IV dose of NT-0231.F provides initial splice rescue at around two weeks, with significant splice rescue around three weeks.