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• PTC Therapeutics Provides Corporate Update and Reports Second Quarter Financial Results

  8/3/2023

  PTC Therapeutics, Inc. announced a corporate update and financial results for the second quarter ending June 30, 2023.

• Ultragenyx Reports Second Quarter 2023 Financial Results and Corporate Update

  8/3/2023

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported its financial results for the quarter ended June 30, 2023 and provided its financial guidance for the year.

• Positive Phase III Results for Genentech’s Ocrevus (ocrelizumab) Twice a Year, 10-Minute Subcutaneous Injection in Patients With Multiple Sclerosis

  7/13/2023

  Genentech, a member of the Roche Group, announced that the Phase III OCARINA II trial evaluating Ocrevus® as a twice a year 10-minute subcutaneous injection met its primary and secondary endpoints in patients with relapsing forms of MS or primary progressive MS.

• Baylor Genetics Announces Epilepsy PanelLatest Panel Analyzes 397 Genes Associated with Several Epilepsy Conditions, Offering an Analysis of Well-studied, Actionable Genes Related to Many Syndromic and Non-Syndromic Epileptic Disorders

  7/11/2023

  Baylor Genetics announced the availability of an Epilepsy Panel and a STAT Epilepsy Panel - with the latter intended for patients with new onset or change in seizure frequency or character.

• ANAVEX Announces Issuance of New U.S. Intellectual Property Compositions Patent for ANAVEX®2-73 (blarcamesine)

  6/1/2023

  Anavex Life Sciences Corp. announced today it was awarded new U.S. Patent No. 11,661,405.

• PTC Therapeutics Announces Strategic Pipeline Prioritization

  5/23/2023

  PTC Therapeutics, Inc. announced the discontinuation of preclinical and early research programs in gene therapy as part of a strategic portfolio prioritization.

• Dyno Therapeutics Launches the Dyno bCap 1™ Capsid Product, a Breakthrough CNS Gene Delivery Vector Created with Generative Artificial Intelligence

  5/19/2023

  Dyno Therapeutics, Inc. today announced the launch of its Dyno bCap 1™ capsid product, a breakthrough CNS-targeted AAV gene delivery vector with best-in-class potential, in a keynote address at the company’s Scientific Symposium at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting.

• Ultragenyx Receives FDA Agreement to Expand Ongoing Global Phase 1/2 Trial Evaluating GTX-102 in Patients with Angelman Syndrome in the U.S.

  5/17/2023

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) has reviewed and agreed to a protocol amendment to the Phase 1/2 study of GTX-102 in pediatric patients with Angelman syndrome.

• Genentech’s BTK Inhibitor Fenebrutinib Significantly Reduced Brain Lesions in People With Relapsing Forms of Multiple Sclerosis

  5/17/2023

  Genentech, a member of the Roche Group, announced positive results from the Phase II FENopta study evaluating investigational oral fenebrutinib in adults with relapsing forms of multiple sclerosis.

• Ultragenyx Reports First Quarter 2023 Financial Results and Corporate Update

  5/4/2023

  Ultragenyx Pharmaceutical Inc. reported its financial results for the quarter ended March 31, 2023 and reaffirmed its financial guidance for 2023.

• HuidaGene Therapeutics Announces 19 Presentations Highlighting Advances in its Gene Therapy Technology Platforms at the 2023 ASGCT Annual Meeting

  5/3/2023

  HuidaGene Therapeutics today announced nineteen (19) presentations on Company's various gene replacement and gene-editing platforms to unlock the full potential of genome medicines at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place in Los Angeles from May 16-20, 2023.

• Genentech Announces Industry-Leading Brain Health Research Collaborations and Latest Data Across Neuroscience Medicines at AAN 2023 Annual Meeting

  4/24/2023

  –– Roche, Genentech and partners embark on global prevalence research, trend-spotting with digital analysis, and telehealth initiatives to assess & address deterrents to brain health to better understand holistic health journeys of patients with neurologic diseases.

• PicnicHealth Announces Continued Real-World Data Strategic Partnership with Roche and Genentech to Advance Neurological Disease Research

  4/24/2023

  PicnicHealth, a patient-centered health technology company, announced its intent to globally expand its partnership with Roche and Genentech to accelerate neurological disease research through investments in real-world data.

• New data presented at AD/PD™2023 show IONIS-MAPT Rx (BIIB080) substantially reduced tau protein in patients with early-stage Alzheimer's disease

  3/29/2023

  Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported that its partner Biogen presented new Phase 1b clinical data showing that IONIS-MAPTRx (BIIB080) reduced soluble tau protein in cerebrospinal fluid (CSF) in a dose-dependent and sustained manner in patients with early-stage Alzheimer's disease (AD).

• Ultragenyx Reports Fourth Quarter and Full Year 2022 Financial Results and Corporate Update

  2/16/2023

  Ultragenyx Pharmaceutical Inc. reported its financial results for the quarter and full year ended December 31, 2022 and reaffirmed its financial guidance for 2023.

• Ultragenyx Reports Preliminary 2022 Revenue; Guidance for 2023 Revenue and Cash Usage; Pipeline Updates and 2023 Milestones

  1/6/2023

  Ultragenyx Pharmaceutical Inc. reported preliminary unaudited 2022 revenue results, cash and investments at year end 2022, and provided 2023 guidance on select key financial metrics including net cash used in operations.

• Exicure, Inc. Announces Termination of AbbVie and Ipsen Collaboration Agreements

  12/14/2022

  Exicure, Inc. an early-stage biotechnology company historically focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets, announced the termination of its collaboration agreements with AbbVie, Inc. and Ipsen BioPharm Limited.

• Anavex Life Sciences Reports Fiscal 2022 Year End Financial Results and Provides Business Update

  11/28/2022

  Anavex Life Sciences Corp. today reported financial results for its fiscal year ended September 30, 2022.

• Ultragenyx Reports Third Quarter 2022 Financial Results and Corporate Update

  11/2/2022

  Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended September 30, 2022 and provided a corporate update for the year.

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  PTC Runs into Latest Huntington's Roadblock with US Trial Pause (Updated)

  10/19/2022

  A Phase II trial studying PTC Therapeutics’ PTC518 in Huntington's disease has been paused in the United States following a request from the FDA for additional data.