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Cerevance Announces Presentation at the 34th International Symposium on ALS/MND
11/28/2023
Cerevance announced a poster presentation at the 34th International Symposium on ALS/MND, taking place in Basel, Switzerland from December 6 – 8, 2023.
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Pasithea Therapeutics Selects PAS-003 Lead Development Candidate, a Humanized Monoclonal Antibody that Targets α5β1 Integrin for the Treatment of both Sporadic and Familial ALS
11/9/2023
Pasithea Therapeutics Corp. announced that it has selected a lead therapeutic candidate for its PAS-003 program, a proprietary humanized monoclonal antibody that targets α5β1 integrin, a protein found to be overexpressed in both human and mouse subjects with amyotrophic lateral sclerosis.
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uniQure Announces Third Quarter 2023 Financial Results and Highlights Recent Company Progress
11/7/2023
uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, reported its financial results for the third quarter of 2023 and highlighted recent progress across its business.
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Voyager Therapeutics Reports Third Quarter 2023 Financial and Operating Results
11/6/2023
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported third quarter 2023 financial and operating results.
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Ionis reports third quarter 2023 financial results
11/2/2023
Ionis Pharmaceuticals, Inc., reported financial results for the third quarter of 2023.
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Ractigen Presents Preclinical Results of Its saRNA Therapeutic Targeting DMD/BMD at the Oligonucleotide Therapeutics Society (OTS) Annual Meeting
11/2/2023
Ractigen Therapeutics presented recent preclinical results at the annual meeting of the Oligonucleotide Therapeutics Society (OTS) in Barcelona, Spain.
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Opinion: The Potential of Neurofilament Light Chain as a Biomarker in Parkinson’s Disease
10/23/2023
While sensitive and specific biomarkers for Alzheimer’s disease have recently taken a leap forward, the Parkinson’s space has lagged behind. Neurofilament Light Chain could change that. -
Recent FDA approvals of novel drugs based on less-than-stellar clinical evidence point to a trend toward regulatory flexibility—particularly in indications with very high unmet need.
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uniQure Announces Strategic Reorganization to Reduce Operating Expenses and Support Advancement of Multiple Clinical-Stage Programs
10/5/2023
uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced a strategic reorganization that will significantly reduce operating expenses while supporting focused execution to rapidly advance multiple clinical-stage programs to proof-of-concept.
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Here’s how some biopharmas have managed to gain funding despite a falloff in investment in the sector. Hint: Positive late-stage data is a key factor.
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Arrowhead Pharmaceuticals to Participate in Upcoming October 2023 Conferences
10/3/2023
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it is scheduled to participate in the following upcoming events.
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After a groundbreaking year in the Alzheimer’s space, Parkinson’s disease researchers express renewed hope based on a greater biological understanding of neurodegeneration.
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The company’s experimental drug for amyotrophic lateral sclerosis reduced the risk of death by 49% compared to the largest U.S. database of previous ALS therapy trials.
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The FDA’s briefing documents found that BrainStorm's BLA submission for its investigational cell therapy for ALS did not demonstrate evidence of effectiveness and that the manufacturing data was “grossly deficient.”
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The FDA will finish September with three action dates and one of the year’s most highly-anticipated advisory committee meetings.
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Aruna Bio Announces Preclinical Results from Pilot Study Evaluating AB126 for the Treatment of Amyotrophic Lateral Sclerosis
9/7/2023
Aruna Bio, Inc. today announced preclinical results from a pilot study evaluating AB126 in a superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) mouse model.
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Aruna Bio Announces Presentation on Treatment Opportunities for its Neural Exosome Platform and Lead Program, AB126 at the Exosome Based Therapeutic Development Summit
9/5/2023
Aruna Bio, Inc., a leader in the development of neural exosome-based therapeutics for the treatment of neurodegenerative diseases, today announced an oral presentation at the Exosome Based Therapeutic Development Summit, being held in Boston, MA from September 5-7, 2023.
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Gain Therapeutics Presents New Preclinical Data Demonstrating a Reduction of Plasma Neurodegeneration Biomarker NfL after Administration of its Drug Candidate GT-02287 in GBA1 Parkinson’s Disease Model
8/28/2023
Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”) today announced preclinical data demonstrating the Company’s lead drug candidate GT-02287 significantly decreased Parkinson’s disease (PD)-associated pathology and improved motor dysfunction in two different preclinical models of Parkinson’s disease.
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Ionis reports second quarter 2023 financial results
8/9/2023
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company"), today reported financial results for the second quarter of 2023.
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Arrowhead Pharmaceuticals Reports Fiscal 2023 Third Quarter Results
8/7/2023
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced financial results for its fiscal third quarter ended June 30, 2023.