Antisense therapy

NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL™ Platform

Tuesday, March 31, 2020 - 12:00pm

ET

Key Points: 
  • ET
    PITTSBURGH, March 31, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq:NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies to address genetic diseases, today announced positive preclinical data from its pharmacokinetics studies in non-human primates (NHPs) and in vitro pharmacodynamics data in patient-derived cell lines.
  • NeuBase Therapeutics, Inc. will discuss these data and next steps for development during a webcasted conference call with slides today, March 31, 2020, at 8:00 a.m.
  • NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides.
  • Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

DGAP-News: Secarna Pharmaceuticals announces the acquisition of LNAplusTM based antisense drug candidates by Lipigon Pharmaceuticals

Monday, March 23, 2020 - 8:01am

Under this agreement, Lipigon acquires certain antisense drug candidates, which were developed with Secarna's proprietary LNAplusTM platform, including the corresponding patent portfolio.

Key Points: 
  • Under this agreement, Lipigon acquires certain antisense drug candidates, which were developed with Secarna's proprietary LNAplusTM platform, including the corresponding patent portfolio.
  • The antisense oligonucleotide candidates targeting the ANGPTL gene family for the treatment of cardiovascular and metabolic diseases were previously generated by Secarna and Lipigon under a target validation and drug discovery collaboration.
  • Going forward, Lipigon will fund and continue the development of the acquired assets and Secarna receives payments customary for such a transaction.
  • Inhibiting the ANGPTL gene family members ANGPTL3 and ANGPTL4 with Secarna's LNAplusTM based antisense oligonucleotides has shown to positively affect plasma lipid levels.

Antisense Therapeutics Announces Appointment of US-based Consultant Medical Director

Thursday, February 27, 2020 - 1:33am

Antisense Therapeutics Limited (ASX:ANP) is pleased to advise of the appointment of Gil Price M.D.

Key Points: 
  • Antisense Therapeutics Limited (ASX:ANP) is pleased to advise of the appointment of Gil Price M.D.
  • William Goolsbee, non-executive director of Antisense Therapeutics, said, It is great news that Dr. Price has agreed to join our team.
  • The addition of Dr. Price as a Consultant Medical Director is an exciting advance for the Company on many fronts.
  • About Antisense Therapeutics Limited (ASX: ANP) is an Australian publicly listed biopharmaceutical company, developing and commercialising antisense pharmaceuticals for large unmet markets.

Akcea Therapeutics to Hold Fourth Quarter and Full Year 2019 Financial Results Webcast

Tuesday, February 18, 2020 - 12:00pm

Akcea Therapeutics, Inc.,a majority-owned affiliate ofIonis Pharmaceuticals, Inc.(NASDAQ: IONS), is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases.

Key Points: 
  • Akcea Therapeutics, Inc.,a majority-owned affiliate ofIonis Pharmaceuticals, Inc.(NASDAQ: IONS), is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious and rare diseases.
  • All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis proprietary antisense technology.
  • In this press release, unless the context requires otherwise, Ionis, Akcea, Company, Companies, we, our, and us refers toIonis Pharmaceuticalsand/orAkcea Therapeutics.
  • Ionis Pharmaceuticals is a trademark ofIonis Pharmaceuticals, Inc., Akcea Therapeutics, TEGSEDIand WAYLIVRAare trademarks ofAkcea Therapeutics, Inc.

Akcea and Ionis report positive topline Phase 2 results of AKCEA-APOCIII-L Rx

Wednesday, January 22, 2020 - 12:05pm

"Based on the positive results from this study, we plan to rapidly pursue development of AKCEA- APOCIII-LRx for familial chylomicronemia syndrome or FCS.

Key Points: 
  • "Based on the positive results from this study, we plan to rapidly pursue development of AKCEA- APOCIII-LRx for familial chylomicronemia syndrome or FCS.
  • AKCEA-APOCIII-LRx was discovered by Ionis and has been co-developed by Akcea and Ionis.
  • All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology.
  • In this press release, unless the context requires otherwise, "Ionis," "Akcea," "Company," "Companies," "we," "our," and "us" refers toIonis Pharmaceuticalsand/or Akcea Therapeutics.

Aro Biotherapeutics and Ionis Enter Licensing and Collaboration Agreement Focused on Cell-Specific Delivery of Antisense Oligonucleotides

Thursday, January 9, 2020 - 12:00pm

Aro Biotherapeutics Company announces it has entered a licensing and collaboration agreement with Ionis Pharmaceuticals, Inc., the leader in RNA-targeted therapeutics, through which Ionis will use Aros CENTYRIN technology to develop targeted cell- and tissue- specific delivery of antisense oligonucleotides (ASOs).

Key Points: 
  • Aro Biotherapeutics Company announces it has entered a licensing and collaboration agreement with Ionis Pharmaceuticals, Inc., the leader in RNA-targeted therapeutics, through which Ionis will use Aros CENTYRIN technology to develop targeted cell- and tissue- specific delivery of antisense oligonucleotides (ASOs).
  • Ionis will leverage this unique technology to develop novel ASO-Centyrin combination drug candidates targeting a variety of diseases with high unmet medical need.
  • Under the terms of the agreement, the two companies have agreed to jointly work towards advancing a defined number of ASO-Centyrin drug conjugates.
  • Aro will be responsible for Centyrin discovery and will collaborate with Ionis to create lead ASO-Centyrin drug conjugates for further development.

Akcea and Ionis Announce Closing of Licensing Agreement with Pfizer Inc. for Investigative Antisense Therapy AKCEA-ANGPTL3-L(Rx)

Tuesday, November 19, 2019 - 9:05pm

AKCEA-ANGPTL3-LRx is an investigational antisense therapy discovered by Ionis and being developed to treat patients with certain cardiovascular and metabolic diseases.

Key Points: 
  • AKCEA-ANGPTL3-LRx is an investigational antisense therapy discovered by Ionis and being developed to treat patients with certain cardiovascular and metabolic diseases.
  • All six drugs were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis proprietary antisense technology.
  • In this press release, unless the context requires otherwise, Ionis, Akcea, Company, Companies, we, our, and us refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
  • Ionis Pharmaceuticals is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics, TEGSEDI and WAYLIVRA are trademarks of Akcea Therapeutics, Inc.

Antisense Therapeutics Announces Additional preliminary data from the ATL1102 Phase II DMD trial presented at the 2019 Action Duchenne International Conference

Monday, November 18, 2019 - 5:00pm

MELBOURNE, Australia, Nov.18, 2019 /PRNewswire/ --Antisense Therapeutics (ASX: ANP/OTC: ATHJY) is pleased to advise that additional preliminary data analyses from the seven patients who have completed their 24 weeks of dosing in the ATL1102 Phase II DMD clinical trial was presented by Dr Ian Woodcock, the Principle Investigator of the ATL1102 Phase II trial at the 2019 Action Duchenne International Conference, Hinkley, UK on 15 November 2019.

Key Points: 
  • MELBOURNE, Australia, Nov.18, 2019 /PRNewswire/ --Antisense Therapeutics (ASX: ANP/OTC: ATHJY) is pleased to advise that additional preliminary data analyses from the seven patients who have completed their 24 weeks of dosing in the ATL1102 Phase II DMD clinical trial was presented by Dr Ian Woodcock, the Principle Investigator of the ATL1102 Phase II trial at the 2019 Action Duchenne International Conference, Hinkley, UK on 15 November 2019.
  • PUL is a functional scale specifically designed for assessing upper limb function in DMD with the aim of reflecting the proximal to distal progression of muscle weakness typically observed in DMD.
  • It includes three domains (shoulder, mid- and distal), each including items exploring activities easily related to activities of daily living that both patients and clinicians regard as relevant.
  • The ATL1102 Phase II DMD trial remains ongoing with dosing in all patients to be completed this month.

The Cell and Gene Therapy Market to Reach Revenues of Over $6.6 billion by 2024 - Market Research by Arizton

Wednesday, November 13, 2019 - 3:00pm

Recently, many innovative and breakthrough cell and gene therapy products have entered the early stage of clinical development.

Key Points: 
  • Recently, many innovative and breakthrough cell and gene therapy products have entered the early stage of clinical development.
  • The US FDA is anticipating more than 200 IND applications for cell & gene therapy products in next few years.
  • Key vendors and pharma giants have placed cell and gene therapy in their strategic area of growth.
  • This is evident from their recent M&As and collaborative partnerships with early-stage drug development companies engaged in the development of cell & gene therapy products.

NeuBase Therapeutics to Present at the Stifel 2019 Healthcare Conference

Tuesday, November 12, 2019 - 2:16pm

NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides.

Key Points: 
  • NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides.
  • The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform allows for the rapid development of targeted drugs, increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of genomic loci or secondary and tertiary RNA structures.
  • Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.