Spinal muscular atrophy

Biogen and Sangamo Announce Global Collaboration to Develop Gene Regulation Therapies for Alzheimer’s, Parkinson’s, Neuromuscular, and Other Neurological Diseases

Retrieved on: 
Thursday, February 27, 2020
Biogen, Spinal muscular atrophy, Life sciences, Industries, Biotechnology

As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance.

Key Points: 
  • As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance.
  • Biogen will then assume responsibility and costs for the investigational new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.
  • Biogen will assume responsibility for GMP manufacturing activities beyond the first clinical trial for each of the first three products.
  • Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.

Biogen and Sangamo Announce Global Collaboration to Develop Gene Regulation Therapies for Alzheimer’s, Parkinson’s, Neuromuscular, and Other Neurological Diseases

Retrieved on: 
Thursday, February 27, 2020
Biotechnology, Health, Genetics, Science, Research, Biogen, Spinal muscular atrophy, Life sciences, Industries, Biotechnology, Biogen, Sangamo Therapeutics

View the full release here: https://www.businesswire.com/news/home/20200227005961/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20200227005961/en/
    As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance.
  • Biogen will then assume responsibility and costs for the investigational new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.
  • Biogen will assume responsibility for GMP manufacturing activities beyond the first clinical trial for each of the first three products.
  • Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies.

Ionis Pharmaceuticals to present at upcoming investor conferences

Retrieved on: 
Thursday, February 27, 2020
Ionis, Spinal muscular atrophy, Antisense RNA

Details on presentation times or changes to presentation dates can be found on the Company's website.

Key Points: 
  • Details on presentation times or changes to presentation dates can be found on the Company's website.
  • A live webcast of the presentations will be available on the Investors & Media section of the Ionis website.
  • 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.

REGENXBIO Reports Fourth Quarter and Full-Year 2019 Financial Results and Operational Highlights

Retrieved on: 
Wednesday, February 26, 2020
Health, Pharmaceutical companies, Spinal muscular atrophy, Novartis, Onasemnogene abeparvovec, Royalty payment

ET

Key Points: 
  • ET
    REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced financial results for the fourth quarter and full year ended December 31, 2019, and recent operational highlights.
  • REGENXBIO expects to provide two-year data from Cohorts 1-3 in the first quarter of 2020.
  • REGENXBIO expects to submit an IND for a Phase II trial for the treatment of DR in the first half of 2020.
  • Recent updates from NAV Technology Licensees include:
    On January 29, 2020, Novartis AG reported fourth quarter 2019 U.S. Zolgensma sales revenue of $186 million, and REGENXBIO recognized royalty revenue of $10.7 million in the fourth quarter of 2019 as a result of these sales.

Ionis provides fourth quarter and full year 2019 financial results

Retrieved on: 
Wednesday, February 26, 2020
Ionis, Income tax in the United States, Tax, Net income, Business, Economy, Articles, Spinal muscular atrophy

Ionis' income tax expense in 2019 was primarily because the Company generated U.S. federal and state taxable income in 2019.

Key Points: 
  • Ionis' income tax expense in 2019 was primarily because the Company generated U.S. federal and state taxable income in 2019.
  • However, because Ionis continues to maintain overall control of Akcea through its voting interest, Ionis reflects the assets, liabilities and results of operations of Akcea in Ionis' consolidated financial statements.
  • Ionis recognized net income attributable to noncontrolling interest in Akcea in 2019 compared to a net loss in 2018.
  • Ionis' net income attributable to Ionis' common stockholders and basic and diluted earnings per share increased in 2019 compared to 2018 primarily due to the significant increase in Ionis' revenue.

Akcea Therapeutics to Present at the Cowen 40th Annual Health Care Conference

Retrieved on: 
Wednesday, February 26, 2020
Spinal muscular atrophy, Biotechnology, Industries, Life sciences, Antisense RNA, Huntington's disease, Ionis Pharmaceuticals, Ionis, Alnylam Pharmaceuticals, NASDAQ Biotechnology Index

A live webcast of the presentation will be available on the Media & Investors section of the Akcea website.

Key Points: 
  • A live webcast of the presentation will be available on the Media & Investors section of the Akcea website.
  • 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.
  • 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.

Adverum Biotechnologies Appoints Angela Thedinga as Chief Technology Officer

Retrieved on: 
Tuesday, February 25, 2020
Branches of biology, Biology, Life sciences, Spinal muscular atrophy, Emerging technologies, Gene delivery, Medical genetics, Molecular biology, AveXis, Adverum Biotechnologies, Novartis, Gene therapy

REDWOOD CITY, Calif., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that Angela Thedinga has been promoted to chief technology officer.

Key Points: 
  • REDWOOD CITY, Calif., Feb. 25, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced that Angela Thedinga has been promoted to chief technology officer.
  • I am pleased to congratulate Angela on her role as CTO, who since joining the company has demonstrated executive leadership across the organization and strategic insight, said Leone Patterson, president and chief executive officer of Adverum Biotechnologies.
  • Before joining Adverum, Ms. Thedinga served as vice president of program management and chief of staff at AveXis, Inc., a gene therapy company acquired by Novartis in 2018.
  • Adverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases.

2020 Geographic Atrophy (GA) Pipeline Insights Report

Retrieved on: 
Wednesday, February 19, 2020
Anatomy, Spinal muscular atrophy, Geographic atrophy, Branches of biology, Atrophy, Medical specialties

Geographic Atrophy (GA) Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Geographic Atrophy (GA) market.

Key Points: 
  • Geographic Atrophy (GA) Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Geographic Atrophy (GA) market.
  • A detailed picture of the Geographic Atrophy (GA) pipeline landscape is provided, which includes the disease overview and Geographic Atrophy (GA) treatment guidelines.
  • The assessment part of the report embraces in-depth Geographic Atrophy (GA) commercial assessment and clinical assessment of the Geographic Atrophy (GA) pipeline products from the pre-clinical developmental phase to the marketed phase.
  • How many Geographic Atrophy (GA) emerging therapies are in early-stage, mid-stage, and late stage of development for the treatment of Geographic Atrophy (GA)?

Ionis Pharmaceuticals to hold 2019 financial results webcast

Retrieved on: 
Wednesday, February 19, 2020
Spinal muscular atrophy, Antisense RNA, Ionis

CARLSBAD, Calif., Feb. 19, 2020 /PRNewswire/ --Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Wednesday, February 26th at 11:30 a.m. Eastern Time to discuss its 2019 financial results and report on pipeline and business progress.

Key Points: 
  • CARLSBAD, Calif., Feb. 19, 2020 /PRNewswire/ --Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Wednesday, February 26th at 11:30 a.m. Eastern Time to discuss its 2019 financial results and report on pipeline and business progress.
  • Click here to access the audio webcast and slides.
  • A webcast replay will remain posted on Ionis' website for a limited time.
  • Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs.

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

Retrieved on: 
Tuesday, February 18, 2020
Life sciences, Spinal muscular atrophy, Biotechnology, Therapeutic gene modulation, Biology, Antisense RNA, Huntington's disease, Ionis Pharmaceuticals, Ionis, Antisense therapy, NASDAQ Biotechnology Index, Alnylam Pharmaceuticals

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.