Argonaute

NeuroSense Therapeutics Reports Positive Preliminary Results in Parkinson's Biomarker Study

Retrieved on: 
Tuesday, May 2, 2023
Neuron, PD, Clinical trial, Pathology, Brain, Biomarker, Movement disorder, Parkinson's disease, Patient, AGO2, LC3, Exosome, Organelle, Therapy, ALS, Argonaute, Pharmaceutical industry, Palladium

This study compared blood samples from 30 healthy individuals to 30 people living with PD by utilizing neuronal derived exosomes to identify modulations in PD-associated biomarkers, including AG02.

Key Points: 
  • This study compared blood samples from 30 healthy individuals to 30 people living with PD by utilizing neuronal derived exosomes to identify modulations in PD-associated biomarkers, including AG02.
  • In the PD biomarker study, NeuroSense observed a statistically significant (p= 0.002) decrease in levels of AGO2 in newly diagnosed PD patients (n=15) when compared to the healthy control group.
  • In the PD biomarker study NeuroSense observed a change in levels of LC3, which decreased in newly diagnosed PD patients (p= 0.034), suggesting an impairment in cellular recycling processes.
  • "Based on these encouraging results, we are now looking to co-develop our PD asset with collaborators that have a core focus on Parkinson's."

Wave Life Sciences Announces Nucleic Acids Research Publication Highlighting Potential Best-in-Class siRNAs Designed with Wave Platform Chemistry

Retrieved on: 
Thursday, April 20, 2023
Drug development, Serum, Liver, Wave, Nucleic Acids Research, MBA, Research, Gene expression, RNA, Biomarker, Doctor of Philosophy, Messenger, Hepatocyte, PN, Small RNA, Gene silencing, N-Acetylgalactosamine, DMD, MD, HSD17B13, Trans-acting siRNA, Pharmacology, Vaccine, Argonaute, RNA interference

CAMBRIDGE, Mass., April 20, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the publication of preclinical data for the company’s small interfering RNA (siRNA) designs in the journal Nucleic Acids Research. Data reported include an in vivo study where a Wave subcutaneously administered, GalNAc-conjugated siRNA maintained approximately 80% silencing of HSD17B13 transcripts for three months in the liver of transgenic mice, while a comparator siRNA format lost silencing activity at the same timepoint. The paper, titled “Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference,” is available here.

Key Points: 
  • This publication demonstrates that by applying our best-in-class oligonucleotide chemistry, including judiciously placed PN backbone chemistry modifications, we can dramatically improve potency and durability for GalNAc-conjugated siRNAs versus comparator siRNA formats.
  • In this publication, Wave’s siRNAs are compared to reference molecules based on siRNAs from a commercial company with a clinically proven track record in RNAi.
  • This suggests there may be ample untapped capacity in endogenous RNAi pathways to further improve siRNA-mediated silencing.
  • PN backbone chemistry modifications in the tested Ttr siRNAs also enhanced Ago2 loading without elevating serum biomarkers for liver dysfunction.

Benitec Biopharma Enrolls First OPMD Subject into the Clinical Development Program

Retrieved on: 
Monday, January 23, 2023
Argonaute, RNA, United, Principal, OPMD, Observation, Oculopharyngeal muscular dystrophy, Child development stages, Benitec Biopharma, Dysphagia, Patient, Oropharyngeal dysphagia, NH, Pharmaceutical industry, Medicine

HAYWARD, Calif., Jan. 23, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “the Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced the enrollment of the first oculopharyngeal muscular dystrophy (OPMD) patient into the OPMD natural history phase of the BB-301 clinical development program.

Key Points: 
  • HAYWARD, Calif., Jan. 23, 2023 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “the Company”), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced the enrollment of the first oculopharyngeal muscular dystrophy (OPMD) patient into the OPMD natural history phase of the BB-301 clinical development program.
  • The OPMD Natural History (NH) Study represents the 6-month pre-treatment observation period for each OPMD subject prior to the administration of BB-301 for the treatment of OPMD-related dysphagia.
  • “Following the initiation of OPMD patient screening at the lead clinical site in the United States in the fourth quarter of 2022, the Principal Investigator of the OPMD NH Study reported high enrollment interest from potential study subjects,” said Jerel A.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.

Benitec Biopharma Releases First Quarter 2023 Financial Results and Provides Operational Update

Retrieved on: 
Thursday, November 10, 2022
IND, CTA, Research, Principal investigator, Principal, Company, GMP, Child development stages, Institutional review board, GDPR, IRB, Disclosure, Benitec Biopharma, Dispute board, RNA, General Data Protection Regulation, U.S. Securities and Exchange Commission, Argonaute, Oculopharyngeal muscular dystrophy, Drug discovery, Dysphagia, Oropharyngeal dysphagia, HDL-Atherosclerosis Treatment Study, HIV disease progression rates, Achievement, United, Privacy, CEC, REB, Phenotype, GLOBE, FIH, Organization, Behavior, Muscle, NASDAQ, Safety, Union, Clinical, Validation, Animal, Natural history, Insurance, Pharmaceutical industry, Medicine, Medical imaging, Mineral wool, Medical device, OPMD, EU

Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.

Key Points: 
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.
  • In the Third Calendar Quarter 2022, Benitec submitted responses to the Central Ethics Committee (CEC) related to minor non-clinical queries.
  • BB-301 Phase 1b/2a Regulatory Updates:
    Investigational New Drug (IND) and Clinical Trial Application (CTA) filings are anticipated in the First Calendar Quarter 2023.
  • Total Revenues for the quarter ended September 30, 2022, were $0 compared to $0 for the quarter ended September 30, 2021.

Benitec Biopharma Inc. Announces Closing of $18 Million Underwritten Public Offering

Retrieved on: 
Friday, September 16, 2022
OPMD, Child development stages, Therapy, RNA interference, Sale, LLC, Company, Private Securities Litigation Reform Act, Risk, U.S. Securities and Exchange Commission, Oculopharyngeal muscular dystrophy, Exercise, Argonaute, Montgomery Street, Nasdaq, Form, RNA, Security (finance), Prospectus, Attention, GLOBE, SEC, Benitec Biopharma

The aggregate gross proceeds to Benitec from the public offering were approximately $17.9 million prior to deducting underwriting discounts, commissions and other estimated offering expenses.

Key Points: 
  • The aggregate gross proceeds to Benitec from the public offering were approximately $17.9 million prior to deducting underwriting discounts, commissions and other estimated offering expenses.
  • JMP Securities, A Citizens Company, acted as sole book-running manager for the offering.
  • Benitec Biopharma Inc. (Benitec or the Company) is a development-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California.
  • This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including with respect to the offering.

Benitec Biopharma Inc. Announces Pricing of $18 Million Underwritten Public Offering

Retrieved on: 
Tuesday, September 13, 2022
OPMD, Child development stages, Therapy, RNA interference, Sale, LLC, Company, Private Securities Litigation Reform Act, Risk, U.S. Securities and Exchange Commission, Oculopharyngeal muscular dystrophy, Exercise, Argonaute, Montgomery Street, Nasdaq, Form, RNA, Security (finance), Prospectus, Attention, GLOBE, SEC, Benitec Biopharma

The offering is expected to close on September 15, 2022, subject to customary closing conditions.

Key Points: 
  • The offering is expected to close on September 15, 2022, subject to customary closing conditions.
  • The aggregate gross proceeds to Benitec from the public offering are expected to be approximately $17.9 million prior to deducting underwriting discounts, commissions and other estimated offering expenses.
  • JMP Securities, A Citizens Company, is acting as sole book-running manager for the offering.
  • Benitec Biopharma Inc. (Benitec or the Company) is a development-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California.

Benitec Biopharma Releases Full Year 2022 Financial Results and Provides Operational Update

Retrieved on: 
Friday, September 2, 2022
Muscle, Safety, Research, Oculopharyngeal muscular dystrophy, Dispute board, Disclosure, Dysphagia, Principal investigator, U.S. Securities and Exchange Commission, GMP, GLOBE, Company, Patient, ICF, Patient Information Advisory Group, FIH, Institutional review board, REB, Phenotype, Argonaute, Annual report, Clinical, Trial of the century, Investigational New Drug, RNA, Form 10-K, NASDAQ, CTA, Oropharyngeal dysphagia, Animal, HDL-Atherosclerosis Treatment Study, Benitec Biopharma, Child development stages, Review, IRB, HIV disease progression rates, CEC, IND, Achievement, Natural history, Validation, Sales, Pharmaceutical industry, Medicine, Medical imaging, Mineral wool, Cryptocurrency, OPMD

HAYWARD, Calif., Sept. 02, 2022 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (Benitec or the Company), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its Fiscal Year ended June 30, 2022.

Key Points: 
  • HAYWARD, Calif., Sept. 02, 2022 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (Benitec or the Company), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its Fiscal Year ended June 30, 2022.
  • The Company has filed its annual report on Form 10-K for the quarter ended June 30, 2022, with the U.S. Securities and Exchange Commission.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.
  • 52-weeks of post-dosing follow-up for conclusive evaluation of the primary and secondary endpoints of the Phase 1b/2a BB-301 treatment study.

Benitec Biopharma Releases Q3 2022 Financial Results

Retrieved on: 
Monday, May 16, 2022
Phenotype, Company, Argonaute, Consultant, U.S. Securities and Exchange Commission, FIH, Principal investigator, GMP, Clinical, NH, Patient, Dysphagia, HDL-Atherosclerosis Treatment Study, File, Insurance, NASDAQ, Pi, ESB Business School, ICF, REB, Research, Natural history, OPMD, IND, Child development stages, Myopathy, Dispute board, CTA, RNA, Safety, Neurology, Charles River Laboratories, CEC, Muscle, Investigational New Drug, GLOBE, Pharmaceutical industry, Medicine, Medical imaging, Civil engineering

HAYWARD, Calif., May 16, 2022 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (Benitec or the Company), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its Fiscal Year Q3 ended March 31, 2022.

Key Points: 
  • HAYWARD, Calif., May 16, 2022 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (Benitec or the Company), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its Fiscal Year Q3 ended March 31, 2022.
  • The Company has filed its quarterly report on Form 10-Q for the quarter ended March 31, 2022, with the U.S. Securities and Exchange Commission.
  • Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma.
  • The Benitec team remains committed to initiating the clinical development program for BB-301 and improving the lives of patients suffering from OPMD.

RNAi Technologies, Companies & Markets, 2028 - ResearchAndMarkets.com

Retrieved on: 
Thursday, November 4, 2021
Health, Genetics, Safety, MicroRNA, RNA interference, Endoribonuclease-prepared siRNA, Degenerative disease, Cancer, Sirna, Tissue, Culture, RNA transfection, Method, Electroporation, Ribonuclease, RNA, Gene, Lists of diseases, Research, Transfection, Gene expression, Table, Gene silencing, Blood, Animal, Company, Argonaute, Genome, Therapy, Publication, Drug discovery, DNA, Bibliography, Cell, Oligonucleotide, Vaccine

The major use of RNAi reagents is in research but it partially overlaps that of drug discovery and therapeutic development.

Key Points: 
  • The major use of RNAi reagents is in research but it partially overlaps that of drug discovery and therapeutic development.
  • Profiles of 168 companies involved in developing RNAi technologies are presented along with 231 collaborations.
  • They are a mix of companies that supply reagents and technologies (nearly half of all) and companies that use the technologies for drug discovery.
  • RNAi microarray has been devised and can be tailored to meet the needs for high throughput screens for identifying appropriate RNAi probes.

Benitec Biopharma to present at H.C. Wainwright's 23rd Annual Global Investment Conference

Retrieved on: 
Monday, September 13, 2021
RNA interference, OPMD, Oculopharyngeal muscular dystrophy, FDA, Intellectual property, Corporation, Marketing, Company, Argonaute, U.S. Securities and Exchange Commission, View, Safety, Therapy, Benitec Biopharma, Investor relations, Multimedia, RNA, Webcast, Research, Pharmaceutical industry

Please visit the link above to register for the presentation which will be presented via webcast during the virtual conference.

Key Points: 
  • Please visit the link above to register for the presentation which will be presented via webcast during the virtual conference.
  • Once your registration is confirmed, you will be prompted to log into the conference website to request a one-on-one meeting with the Company.
  • Benitec Biopharma, Inc. ("Benitec" or the "Company") is a development-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California.
  • Media & Investor Relations Contact:
    CEO of M Group Strategic Communications (for Benitec Biopharma, Inc.)
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