Therapeutic gene modulation

Ionis Chief Scientific Officer Frank Bennett receives Lifetime Achievement Award from Oligonucleotide Therapeutics Society

Retrieved on: 
Thursday, October 1, 2020
Therapeutic gene modulation, Antisense RNA, Life sciences, Biology, Spinal muscular atrophy, Genetics, Huntington's disease, Ionis Pharmaceuticals, Ionis, Antisense therapy, C. Frank Bennett

CARLSBAD, Calif., Oct. 1, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that Frank Bennett, Ph.D., Ionis' executive vice president and chief scientific officer, has been awarded the 2020 Lifetime Achievement Award by the Oligonucleotide Therapeutics Society (OTS).

Key Points: 
  • CARLSBAD, Calif., Oct. 1, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today that Frank Bennett, Ph.D., Ionis' executive vice president and chief scientific officer, has been awarded the 2020 Lifetime Achievement Award by the Oligonucleotide Therapeutics Society (OTS).
  • A founding member of Ionis, Dr. Bennett is responsible for continuing to advance antisense technology and expanding Ionis' drug discovery platform.
  • He is also the franchise leader for neurological programs at Ionis.
  • "I am deeply honored and humbled to be recognized by the Oligonucleotide Therapeutics Society with the Lifetime Achievement Award.

Alnylam to Webcast Presentation at Chardan Virtual 4th Annual Genetic Medicines Conference

Retrieved on: 
Monday, September 28, 2020
Health, Infectious diseases, Genetics, Other Health, General Health, Pharmaceutical, Biotechnology, Alnylam Pharmaceuticals, Therapeutic gene modulation, Biotechnology, Life sciences, Companies

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Chardan Virtual 4th Annual Genetic Medicines Conference on Monday, October 5, 2020 at 4:15 pm ET via webcast.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Chardan Virtual 4th Annual Genetic Medicines Conference on Monday, October 5, 2020 at 4:15 pm ET via webcast.
  • A live audio webcast of the presentation will be available on the Investors section of the Companys website at www.alnylam.com/events .
  • A replay will be available on the Alnylam website within 48 hours after the event.
  • Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development.

WAYLIVRA▼® (volanesorsen), the First and Only Therapy for FCS, an Ultra-Rare and Life-Threatening Condition, to Be Available on the NHS in England

Retrieved on: 
Friday, September 18, 2020
Biotechnology, Pharmaceutical, Health, Clinical trials, Antisense RNA, Therapeutic gene modulation, Health in England, National Health Service, EAMS, NHS Scotland, Volanesorsen, Branches of biology, volanesorsen, FCS

This important milestone will allow patients with this severely debilitating condition to access treatment on the NHS, drastically improving their quality of life.

Key Points: 
  • This important milestone will allow patients with this severely debilitating condition to access treatment on the NHS, drastically improving their quality of life.
  • Akcea Therapeutics initially made volanesorsen available to patients with FCS through the Early Access to Medicines Scheme (EAMS).
  • Since initiation of the EAMS, 22 patients with FCS have been treated with volanesorsen, at no cost to the NHS.
  • Volanesorsen will be the second treatment that Akcea Therapeutics has made available through the NHS in the last year.

Alnylam Highlights Prospects for Building an Industry-Leading ATTR Amyloidosis Franchise at RNAi Roundtable

Retrieved on: 
Thursday, September 3, 2020
Oncology, FDA, Health, Clinical trials, Pharmaceutical, Biotechnology, Branches of biology, Biotechnology, Biology, Therapeutic gene modulation, Alnylam Pharmaceuticals, RNA, Gene expression, RNA interference, Familial amyloid polyneuropathy, ONPATTRO® (patisiran), hATTR Amyloidosis, RNAi, Alnylam Pharmaceuticals

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, will host an RNAi Roundtable webinar today at 12:00 p.m.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, will host an RNAi Roundtable webinar today at 12:00 p.m.
  • We believe we have the potential to build an industry-leading franchise of medicines for the treatment of ATTR amyloidosis, and we look forward to highlighting our progress in our RNAi Roundtable later today.
  • Vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis, is currently being evaluated in the HELIOS Phase 3 program.
  • Todays RNAi Roundtable webinar can be accessed at www.alnylam.com/events or on the Capella section of the Alnylam website.

Alnylam to Webcast Presentations at Upcoming September Investor Conferences

Retrieved on: 
Wednesday, September 2, 2020
Biotechnology, Infectious diseases, Health, Genetics, Pharmaceutical, Alnylam Pharmaceuticals, Therapeutic gene modulation, Biotechnology, Companies, LinkedIn, Life sciences

A replay will be available on the Alnylam website within 48 hours after each event.

Key Points: 
  • A replay will be available on the Alnylam website within 48 hours after each event.
  • Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.
  • Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development.
  • For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn .

Ionis Pharmaceuticals to hold cardio-renal franchise webcast

Retrieved on: 
Wednesday, August 26, 2020
Branches of biology, Biology, Therapeutic gene modulation, Life sciences, RNA, Molecular biology, Ionis, Sense, Ionis Pharmaceuticals, Antisense therapy

A webcast replay will be available for a limited time at the same address.

Key Points: 
  • A webcast replay will be available for a limited time at the same address.
  • As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective.
  • Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs.
  • To learn more about Ionis visit www.ionispharma.com and follow us on twitter @ionispharma.

Akcea and Ionis to Present Phase 2 Results of AKCEA-APOCIII-L(Rx) in Late-Breaking Presentation at ESC Congress 2020

Retrieved on: 
Tuesday, August 18, 2020
Therapeutic gene modulation, Antisense RNA, Spinal muscular atrophy, Life sciences, Huntington's disease, Ionis Pharmaceuticals, Biology, Genetics, Antisense therapy, Ionis

AKCEA-APOCIII-LRx was discovered by Ionis and has been co-developed by Akcea and Ionis.

Key Points: 
  • AKCEA-APOCIII-LRx was discovered by Ionis and has been co-developed by Akcea and Ionis.
  • Akcea and Ionis intend to pursue development in familial chylomicronemia syndrome, or FCS, and are considering pursuing development in additional indications.
  • 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.

Blackstone and Alnylam Close $150 Million R&D Financing to Advance RNAi Therapeutics for Cardiovascular Disease

Retrieved on: 
Monday, August 17, 2020
Infectious diseases, Finance, Genetics, Professional services, Cardiology, Biotechnology, Pharmaceutical, Health, Science, Other Science, Biotechnology, Branches of biology, Biology, Alnylam Pharmaceuticals, Therapeutic gene modulation, RNA, Gene expression, RNA interference, RNAi, Alnylam Pharmaceuticals, Blackstone Life Sciences

Blackstone (NYSE: BX) and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the closing of the R&D funding component of the companies previously announced $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics.

Key Points: 
  • Blackstone (NYSE: BX) and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the closing of the R&D funding component of the companies previously announced $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics.
  • Under the terms of the agreement, Alnylam will receive up to $150 million from Blackstone Life Sciences for the development of Alnylams cardiometabolic disease programs vutrisiran and ALN-AGT.
  • By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality.
  • Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.

Alnylam Announces Planned Transition of Barry Greene, President

Retrieved on: 
Thursday, August 6, 2020
Biotechnology, Pharmaceutical, Genetics, Health, Alnylam Pharmaceuticals, Therapeutic gene modulation, Biotechnology, Life sciences, Companies, RNAi, Alnylam Pharmaceuticals

It has been my tremendous privilege to serve as President of Alnylam for such a long period of time.

Key Points: 
  • It has been my tremendous privilege to serve as President of Alnylam for such a long period of time.
  • I will continue to support Alnylam in this transition and have no doubts about the Companys future prospects as a top tier biopharmaceutical company.
  • I know that I speak for everyone at Alnylam in thanking Barry for his exceptional contributions and dedication to the Company, to patients and to our employees.
  • We are, however, pleased that he will remain involved with Alnylam as a consultant sharing his in-depth knowledge, extensive network and experience.

DGAP-News: Secarna Pharmaceuticals and Evotec form strategic partnership in the field of antisense therapy

Retrieved on: 
Wednesday, August 5, 2020
Branches of biology, Biology, Life sciences, Evotec, Therapeutic gene modulation, Antisense therapy, Sense, Oligonucleotide, TecDAX, MDAX, Secarna's proprietary drug discovery platform, LNAplusTM, Secarna Pharmaceuticals GmbH & Co. KG, Evotec SE

Munich/Martinsried and Hamburg, Germany, August 05, 2020 - Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) and the leading independent European antisense drug discovery company, Secarna Pharmaceuticals GmbH & Co. KG ("Secarna"), today announced a strategic partnership in the field of antisense oligonucleotide ("ASO")-based therapeutics.

Key Points: 
  • Munich/Martinsried and Hamburg, Germany, August 05, 2020 - Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) and the leading independent European antisense drug discovery company, Secarna Pharmaceuticals GmbH & Co. KG ("Secarna"), today announced a strategic partnership in the field of antisense oligonucleotide ("ASO")-based therapeutics.
  • Using state-of-the-art bioinformatics (OligofyerTM), Secarna's antisense molecules are precisely engineered to specifically bind to the targeted RNA of the gene of interest.
  • With their framework agreement, Evotec and Secarna have laid the foundation for a long-term platform collaboration spanning across a number of targets and indications.
  • Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "This strategic partnership with Secarna further complements Evotec's multimodality platform.