Therapeutic gene modulation

Progenity and Ionis Pharmaceuticals Enter into Agreement to Evaluate Progenity’s Ingestible Oral Biotherapeutics Technology for Delivery of Antisense Therapies

Retrieved on: 
Tuesday, April 6, 2021
Branches of biology, Biology, Life sciences, Biotechnology, Molecular biology, Therapeutic gene modulation, RNA, Applied genetics, Antisense therapy, Sense, Ionis, Glucagon-like peptide-1

Ionis novel antisense therapies are designed to target mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased.

Key Points: 
  • Ionis novel antisense therapies are designed to target mRNA in a highly specific manner, so that the amount of disease-causing protein is dramatically decreased.
  • Were excited to work with Ionis, a leader in nucleic-acid-based biotherapeutics, to collectively evaluate the ODBS platform for the oral delivery of antisense therapies, said Harry Stylli, PhD, CEO, chairman of the board and co-founder of Progenity.
  • We believe the OBDS platform shows promise to transform the systemic delivery of diverse biotherapeutics via oral administration.
  • Progenity is developing an internal pipeline including PGN-OB1, an oral version of adalimumab and PGN-OB2, an oral version of a GLP-1 analog.

Alnylam to Webcast Presentation at Stifel 3rd Annual CNS Day

Retrieved on: 
Monday, March 29, 2021
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Branches of biology, Biotechnology, Biology, Alnylam Pharmaceuticals, Therapeutic gene modulation, RNA, RNA interference

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Stifel 3rd Annual CNS Day on Thursday, April 1, 2021 at 11:00 am ET.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Stifel 3rd Annual CNS Day on Thursday, April 1, 2021 at 11:00 am ET.
  • 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.
  • Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.

Marinus Pharmaceuticals Appoints Dr. Saraswathy (Sara) Nochur to Board of Directors

Retrieved on: 
Wednesday, March 24, 2021
Biotechnology, Pharmaceutical, Health, Clinical trials, Alnylam Pharmaceuticals, Therapeutic gene modulation, Biotechnology, Companies, Life sciences

Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the appointment of Saraswathy (Sara) Nochur, Ph.D., to its Board of Directors.

Key Points: 
  • Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders, today announced the appointment of Saraswathy (Sara) Nochur, Ph.D., to its Board of Directors.
  • Dr. Nochur currently serves as Chief Diversity, Equity and Inclusion Officer at Alnylam Pharmaceuticals in Cambridge, MA.
  • Sara is an excellent addition to the board, and we are confident that her extensive background will complement the skill sets of our current board members, said Nicole Vitullo, Director, Chairman of the Board at Marinus.
  • Marinus Pharmaceuticals, Inc. is a pharmaceutical company dedicated to the development of innovative therapeutics to treat rare seizure disorders.

Alnylam Issues Inaugural Corporate Responsibility Summary

Retrieved on: 
Thursday, March 11, 2021
Pharmaceutical, Health, Environment, Alnylam Pharmaceuticals, Therapeutic gene modulation, Biotechnology, Companies, Life sciences, RNAi, Alnylam Pharmaceuticals

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today issued its first Corporate Responsibility (CR) Summary, formalizing the management, tracking and communication of its environmental, social and governance (ESG) standards worldwide.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today issued its first Corporate Responsibility (CR) Summary, formalizing the management, tracking and communication of its environmental, social and governance (ESG) standards worldwide.
  • The summary highlights Alnylams commitments to sustainable business practices and demonstrated focus on its people, the communities it intends to serve, and the planet.
  • The summary, available online at Alnylam.com, features case studies and analyses that illustrate the company's CR-driven philosophy, accomplishments, aspirations and areas of future focus.
  • To read more about Alnylams CR summary, framework and key priorities, download Corporate Responsibility at Alnylam here .

MDA Celebrates FDA Approval of Amondys 45 for Treatment of DMD Amenable to Exon 45 Skipping

Retrieved on: 
Thursday, February 25, 2021
Muscular dystrophy, Therapeutic gene modulation, Antisense RNA, Branches of biology, Biotechnology, Biology, Orphan drugs, Eteplirsen, Dystrophin, Viltolarsen, Duchenne muscular dystrophy

Amondys 45 is designed to treat a third subset of patients with DMD, specifically those with a mutation amenable to skipping of exon 45.

Key Points: 
  • Amondys 45 is designed to treat a third subset of patients with DMD, specifically those with a mutation amenable to skipping of exon 45.
  • Approval of Amondys 45 represents another significant step forward in the development of therapies for DMD that target the root cause of the disease.
  • It is estimated that up to 8% of patients with DMD have mutations amenable to treatment with Amondys 45.
  • Just as individuals with DMD caused by a mutation that would be amenable to skipping exon 51 could benefit from treatment with Exondys 51, those with DMD caused by a mutation that would be impacted by skipping exon 45 potentially could benefit from treatment with Amondys 45.

Alnylam to Webcast Presentations at Upcoming March Investor Conferences

Retrieved on: 
Monday, February 22, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Companies, Alnylam Pharmaceuticals, Therapeutic gene modulation, Biotechnology, 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 .

Alnylam to Webcast Presentation at 10th Annual SVB Leerink Global Healthcare Conference

Retrieved on: 
Thursday, February 18, 2021
Biotechnology, Pharmaceutical, Genetics, Health, Biotechnology, Branches of biology, Companies, Alnylam Pharmaceuticals, Therapeutic gene modulation, RNA, SVB Leerink, RNA interference

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the 10th Annual SVB Leerink Global Healthcare Conference on Thursday, February 25, 2021 at 11:20 am ET.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the 10th Annual SVB Leerink Global Healthcare Conference on Thursday, February 25, 2021 at 11:20 am ET.
  • A live audio webcast of the call 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.
  • Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform.

Alnylam to Webcast Conference Call Discussing Fourth Quarter and Full Year 2020 Financial Results

Retrieved on: 
Thursday, January 28, 2021
Biotechnology, Pharmaceutical, Health, Oncology, Biotechnology, Branches of biology, Biology, Alnylam Pharmaceuticals, Therapeutic gene modulation, RNA, NASDAQ, RNA interference, Conference call

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it will report financial results for the fourth quarter and year ending December 31, 2020 on Thursday, February 11, 2021, before the U.S. financial markets open.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that it will report financial results for the fourth quarter and year ending December 31, 2020 on Thursday, February 11, 2021, before the U.S. financial markets open.
  • Management will provide an update on the Company and discuss fourth quarter and year-end 2020 results as well as expectations for the future via conference call on Thursday, February 11, 2021 at 8:30 am ET.
  • To access the call, please dial 877-312-7507 (domestic) or +1-631-813-4828 (international) five minutes prior to the start time and refer to conference ID 6180123.
  • A replay of the call will be available beginning at 11:30 am ET on the day of the call.

Alnylam Launches “Alnylam P5x25” Strategy for Planned Transition to a Top Five Biotech in Market Capitalization Over Next Five Years

Retrieved on: 
Sunday, January 10, 2021
Research, General Health, Pharmaceutical, Optical, Infectious diseases, Genetics, Clinical trials, Science, Cardiology, Biotechnology, Medical Supplies, Health, Branches of biology, Biology, Biotechnology, Alnylam Pharmaceuticals, Therapeutic gene modulation, RNA, Neurological disorders, Transthyretin, Familial amyloid polyneuropathy, Patisiran, Amyloidosis, RNA interference

Alnylam P5x25 extends the Companys decade-long heritage of providing longer term, 5-year business strategy guidance, the most recent of which was known as Alnylam 2020.

Key Points: 
  • Alnylam P5x25 extends the Companys decade-long heritage of providing longer term, 5-year business strategy guidance, the most recent of which was known as Alnylam 2020.
  • The Companys Alnylam P5x25 strategy is aimed at Alnylams transition to a top 5 biotech company, as measured by market capitalization, over the next 5 years.
  • ONPATTRO (patisiran), a commercial-stage RNAi therapeutic targeting transthyretin (TTR) for the treatment of polyneuropathy in adult patients with hATTR amyloidosis.
  • **The Alnylam P5x25 metrics on Patients, Products, and Pipeline include proprietary and partnered products and programs.

Ionis appoints Eugene Schneider, M.D., as executive vice president and chief clinical development officer

Retrieved on: 
Thursday, January 7, 2021
Antisense RNA, Spinal muscular atrophy, Huntington's disease, Ionis Pharmaceuticals, Ionis, Therapeutic gene modulation

CARLSBAD, Calif., Jan. 7, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.(NASDAQ: IONS) today announced the appointment ofEugene Schneider, M.D.,as executive vice president and chief clinical development officer.

Key Points: 
  • CARLSBAD, Calif., Jan. 7, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.(NASDAQ: IONS) today announced the appointment ofEugene Schneider, M.D.,as executive vice president and chief clinical development officer.
  • In this role, Dr. Schneider will oversee clinical development of novel antisense medicines across all of Ionis' therapeutic franchises.
  • He will report to Richard S. Geary, Ph.D., Ionis' executive vice president and chief development officer, and serve on the company's executive team.
  • Since joining Ionis in 2013, Dr. Schneider has played a key role in more than 20 clinical trials conducted in the U.S. and globally.