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Inhibikase Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Activity

Retrieved on: 
Wednesday, March 27, 2024
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The Meeting Minutes confirmed that the 505(b)(2) pathway appears to be appropriate for approval of IkT-001Pro.

Key Points: 
  • The Meeting Minutes confirmed that the 505(b)(2) pathway appears to be appropriate for approval of IkT-001Pro.
  • Inhibikase will host a conference call and webcast to discuss its full-year 2023 financial results and business highlights tomorrow, March 28, 2024, at 8:00am ET.
  • The conference call can be accessed by dialing 1-877-407-0789 (United States) or 1-201-689-8562 (International) and referencing Inhibikase Therapeutics.
  • After the live webcast, the event will be archived on Inhibikase’s website for approximately 90 days after the call.

Filippo Berio announces exclusive partnership with Alzheimer's Drug Discovery Foundation, supporting critical research and brain health

Retrieved on: 
Monday, April 8, 2024
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LYNDHURST, N.J., April 8, 2024 /PRNewswire/ -- Filippo Berio, one of the nation's top-selling and longstanding olive oil brands, has established an exclusive partnership with the Alzheimer's Drug Discovery Foundation to raise awareness for Alzheimer's disease and contribute crucial funds to support groundbreaking research aimed at finding a cure for Alzheimer's and related dementias. The collaboration is a natural fit, given the growing body of research that indicates properties in olive oil may not only play a pivotal role in cardiovascular health but also provide important benefits to brain health. The partnership aims to increase overall awareness of lifestyle changes, including a healthy diet, that may positively affect brain health, which can help consumers to make healthier, informed choices.

Key Points: 
  • The collaboration is a natural fit, given the growing body of research that indicates properties in olive oil may not only play a pivotal role in cardiovascular health but also provide important benefits to brain health.
  • The partnership aims to increase overall awareness of lifestyle changes, including a healthy diet, that may positively affect brain health, which can help consumers to make healthier, informed choices.
  • Studies indicate that consuming olive oil may have protective effects on the brain and reduce the risk of Alzheimer's."
  • This will be the second consecutive year Filippo Berio is supporting one of the ADDF's signature fundraising events.

Orphan designation: rovatirelin Treatment of spinocerebellar ataxia, 11/11/2022 Positive

Retrieved on: 
Tuesday, April 9, 2024
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Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of spinocerebellar ataxia in the European Union on 11 November 2022.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Retrieved on: 
Tuesday, April 9, 2024
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Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Key Points: 


Orphan designation: Glycyl-L-2-methylprolyl-L-glutamic acid Treatment of fragile X syndrome, 28/07/2015 Positive

Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy

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Wednesday, March 20, 2024
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TOKYO, LONDON and BOSTON, March 20, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the details of its U.S. commercial launch of Lenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, the first FDA-approved therapy for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile, (PSEJ), or early symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD).

Key Points: 
  • Prior to Lenmeldy, there were no treatment options in the U.S. for early-onset MLD beyond supportive and end-of-life care.
  • This approach has the potential to restore enzymatic function to stop or slow disease progression with a single treatment.
  • Utilizing results from such studies, a multi-stakeholder working group is finalizing a nomination to add MLD to the U.S.
  • Based on current timelines and assumptions, Orchard Therapeutics expects the nomination will be submitted in mid-year 2024.

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.

Retrieved on: 
Monday, March 18, 2024
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“MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.

Key Points: 
  • “MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
  • This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy.
  • It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA.
  • Orchard Therapeutics will provide more details about the launch of Lenmeldy in the U.S. through a separate announcement this week.

Clene Announces Peer-Reviewed Publication Characterizing the Protein Corona of the Investigational Neurodegenerative Disease Drug, CNM-Au8®

Retrieved on: 
Friday, March 15, 2024
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Simultaneously, the depletion of opsonin-based proteins in the corona extends their circulation time in the blood, as observed clinically.

Key Points: 
  • Simultaneously, the depletion of opsonin-based proteins in the corona extends their circulation time in the blood, as observed clinically.
  • “When we invented the electro-crystal-chemical method to grow clean-surfaced, highly faceted gold nanocrystals, we certainly had catalytic optimization in mind.
  • However, we also understood that the protein corona can control the location of our nanocrystals in vivo.
  • A Phase 3 registrational clinical trial of CNM-Au8 for the treatment of the progressive neurodegenerative disease, amyotrophic lateral sclerosis, is presently planned to launch in 2024.

Voyager Therapeutics Announces Appointment of Toby Ferguson as Chief Medical Officer

Retrieved on: 
Wednesday, March 13, 2024
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LEXINGTON, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the appointment of Toby Ferguson, M.D., Ph.D., as Chief Medical Officer (CMO), effective March 25, 2024.

Key Points: 
  • LEXINGTON, Mass., March 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today announced the appointment of Toby Ferguson, M.D., Ph.D., as Chief Medical Officer (CMO), effective March 25, 2024.
  • During his tenure, he built and developed teams focused on neuromuscular and movement disorders, overseeing strategy for these areas across Biogen R&D.
  • Prior to joining Biogen, Ferguson was Assistant Professor of Neurology, Shriners Pediatric Research Center and Temple University School of Medicine.
  • Voyager represents for me an extraordinary opportunity to fulfill this mission.”

Novocure Announces 20 Presentations On Tumor Treating Fields, Highlighting Preclinical Effects in Pancreatic Cancer and Immune Effects, at American Association for Cancer Research (AACR) Annual Meeting 2024

Retrieved on: 
Friday, April 5, 2024
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Novocure (NASDAQ: NVCR) today announced 20 presentations on Tumor Treating Fields (TTFields) therapy will be delivered at the American Association for Cancer Research (AACR) Annual Meeting 2024, to be held April 5 to 10 in San Diego.

Key Points: 
  • Novocure (NASDAQ: NVCR) today announced 20 presentations on Tumor Treating Fields (TTFields) therapy will be delivered at the American Association for Cancer Research (AACR) Annual Meeting 2024, to be held April 5 to 10 in San Diego.
  • Presentations from Novocure-sponsored and partner programs include:
    Tumor Treating Fields (TTFields) targeted self assembling nanoparticles for pancreatic cancer treatment: In vitro and in vivo assessment.
  • Tumor Treating Fields (TTFields) induce an anti-tumor immune response in a pancreatic cancer mouse model.
  • Tumor Treating Fields (TTFields) show efficacy in Triple-Negative breast cancer (TNBC) cells alone and in combination with PARP inhibitor Talazoparib.

New Findings From Sunbird Bio Reveal Significant Potential of Specific Tau Proteins to Serve as Blood-Based Biomarker for Alzheimer’s Disease

Retrieved on: 
Monday, March 25, 2024
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“These results validate the potential of EV-bound tau proteins to serve as reliable blood-based biomarkers in accurately detecting Alzheimer’s disease,” said John McDonough, executive chair and CEO of Sunbird Bio.

Key Points: 
  • “These results validate the potential of EV-bound tau proteins to serve as reliable blood-based biomarkers in accurately detecting Alzheimer’s disease,” said John McDonough, executive chair and CEO of Sunbird Bio.
  • “This confirmed correlation between EV-bound tau in the blood and tau tangles in the brain combined with the early validation of our new tau assays is incredibly exciting.
  • The Sunbird Bio study being shared at Tau2024 evaluated the potential of EV-bound tau proteins in the blood as a method of directly detecting the tau proteins that aggregate in the brain.
  • They then used Sunbird Bio’s new proprietary tau assays to distinguish between EV-bound and unbound soluble forms of tau in plasma.