Lilly Acquires Prevail and Gene Therapy for Neuronopathic Gaucher – Gaucher Disease News

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• Prevail lands on a Lilly pad December 15, 2020 # # # # 2020 has been a dreadful year for most of the world – burdened by the outbreak and consequences of COVID-19. Despite this, there has been a steady stream of biotech acquisitions related to Parkinson’s which have helped to keep morale high in the PD research community. In October alone, we saw the Portuguese pharmaceutical company Bial…
• Monthly Research Review – December 2020 December 31, 2020   At the end of each month the SoPD writes a post which provides an overview of some of the major pieces of Parkinson’s-related research that were made available during December 2020. The post is divided into seven parts based on the type of research: Basic biology Disease mechanism Clinical research New clinical trials Clinical trial news Conferences/lectures Other news…
• Bayer doubles down on Parkinson’s? December 10, 2020 # # # # News today of two biotech companies merging did not cause much of a ripple in the media, but the wider implications of the move are rather significant for Parkinson’s. Today it was announced that Brain Neurotherapy Bio (BNB) is going to merge with Asklepios Biopharmaceutical (aka AskBio). BNB are currently clinically testing a GDNF gene therapy…
• The road ahead: 2020 January 5, 2020 Here at the SoPD, we are primarily interested in disease modification for Parkinson’s. While there is a great deal of interesting research exploring the causes of the condition, novel symptomatic therapies, and other aspects of Parkinson’s, my focus is generally on the science seeking to slow, stop or reverse the condition. At the start of each year, it is a…
• Monthly Research Review – September 2019 September 30, 2019   At the end of each month the SoPD writes a post which provides an overview of some of the major pieces of Parkinson’s-related research that were made available during September 2019. The post is divided into seven parts based on the type of research: Basic biology Disease mechanism Clinical research New clinical trials Clinical trial news Other news Review…
• 2019: Year in review December 31, 2019   In this end-of-year post, we review the Parkinson’s research that caught our attention at SoPD HQ in 2019. Month-by-month we will briefly discuss some of the major pieces of research/announcements that have defined the year and advanced our understanding of Parkinson’s. The list is based on nothing more than the author’s personal opinion – apologies to any researchers who…
• Monthly research review – October 2019 October 31, 2019   At the end of each month the SoPD writes a post which provides an overview of some of the major pieces of Parkinson’s-related research that were made available during October 2019. The post is divided into seven parts based on the type of research: Basic biology Disease mechanism Clinical research New clinical trials Clinical trial news Other news Review…
• Prevail Therapeutics Provides Program Update on PR001 in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease - GlobeNewswire September 10, 2019 Prevail Therapeutics Provides Program Update on PR001 in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease  GlobeNewswireNEW YORK, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (NASDAQ: PRVL), a biotechnology company developing AAV-based gene ...
• 2020: Year in Review January 1, 2021 # # # # In this end-of-year post, we review the Parkinson’s research that caught our attention at SoPD HQ in 2020. Month-by-month we will briefly discuss some of the major pieces of research/ announcements that have defined the year and advanced our understanding of Parkinson’s. The list is based on nothing more than the author’s personal opinion – apologies…
• Venture Capitalist Launches Foundation, Co-Founds New Biotech to Find Cure for His Parkinson’s August 11, 2017 Jonathan Silverstein’s two decades as a healthcare venture capitalist gave him the solid background he needed to be a success in his career. But it didn’t quite prepare him for his diagnosis last year at age 49 with Parkinson’s disease with a rare mutation in the GBA gene. Although overwhelmed by the statistics — there is no cure for Parkinson’s and…
• Prevail Therapeutics Provides Program Update on PR001 in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease - StreetInsider.com September 10, 2019 Prevail Therapeutics Provides Program Update on PR001 in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease  StreetInsider.comGet instant alerts when news breaks on your stocks. Claim your 2-week free trial to StreetInsider Premium here. NEW YORK, Sept. 10, 2019 (GLOBE ...
• Enrollment Ongoing in PROPEL Trial for Prevail’s Gene Therapy PR001 March 9, 2020 Enrollment in the Phase 1/2 PROPEL trial, evaluating the safety, tolerability, and early efficacy of PR001 — a one-time gene therapy — for Parkinson’s disease associated with mutations in the GBA1 gene, is progressing at two centers in New York and patient dosing is continuing. Prevail Therapeutics, which is developing the therapy, expects to present early interim results in the second…
• Prevail’s Gene Therapy Candidate PR001 Granted FDA Fast Track Status July 10, 2019 The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Prevail Therapeutics’ lead gene therapy candidate, PR001, for the treatment of people with Parkinson’s disease associated with GBA1 gene mutations. Fast Track status will support and expedite the clinical development, regulatory review, and potential marketing approval of PR001. The FDA’s decision follows its acceptance of Prevail’s Investigational New…
• Say it with me: Farn-e-syl-trans-fer-ase October 24, 2019 ng   Not a week goes by without some new peice of research suggesting yet another biological mechanism that could be useful in slowing or stopping Parkinson’s. This week researchers in Chicago reported that pharmacologically inhibiting a specific enzyme – farnesyltransferase – may represent a novel means of boosting waste disposal and helping stressed cells to survive. A number of…
• Potential One-time Gene Therapy for Parkinson’s Linked to GBA Mutations to Enter Clinical Trial June 11, 2019 A potential gene therapy for Parkinson’s disease associated with mutations in the GBA1 gene, PR001, will move into clinical testing in patients after the U.S. Food and Drug Administration (FDA) accepted an application for the therapy, Prevail Therapeutics announced. FDA acceptance of the company’s Investigational New Drug (IND) application allows Prevail to initiate a Phase 1/2 clinical trial assessing PR001’s safety…
• Knowledge and attitudes of Parkinson disease risk in the Gaucher population. October 31, 2019 Knowledge and attitudes of Parkinson disease risk in the Gaucher population. J Genet Couns. 2019 Oct 30;: Authors: Zaretsky L, Zeid N, Naik H, Alcalay RN, Balwani M Abstract Individuals with Gaucher disease have an increased risk of developing Parkinson's disease as compared to the general population. Though the association is well-known in the scientific community, it is unclear if…
• Prevail Therapeutics Provides Program Update on PR001 in Parkinson’s Disease with GBA1 Mutations and Neuronopathic Gaucher Disease - Yahoo Finance September 10, 2019 Prevail Therapeutics Provides Program Update on PR001 in Parkinson’s Disease with GBA1 Mutations and Neuronopathic Gaucher Disease  Yahoo FinancePrevail Therapeutics Inc. (NASDAQ: PRVL), a biotechnology company developing AAV-based gene therapies for patients with neurodegenerative disorders, ...
• Prevail Therapeutics Provides Program Update on PR001 in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease - Nasdaq September 10, 2019 Prevail Therapeutics Provides Program Update on PR001 in Parkinson's Disease with GBA1 Mutations and Neuronopathic Gaucher Disease  NasdaqNEW YORK, Sept. 10, 2019-- Prevail Therapeutics Inc., a biotechnology company developing AAV-based gene therapies for patients with.
• Early Trial for Prevail’s One-time Gene Therapy PR001 to Start Soon September 12, 2019 Prevail Therapeutics will soon start its Phase 1/2 clinical trial evaluating the safety, tolerability, and early efficacy of its one-time gene therapy — called PR001 — for Parkinson’s disease associated with mutations in the GBA1 gene. The trial is expected to enroll up to 16 people with confirmed GBA1 mutations. Participants will be randomly assigned to receive two escalating doses of PR001, or…
• Glucocerebrosidase and its relevance to Parkinson disease. August 30, 2019 Glucocerebrosidase and its relevance to Parkinson disease. Mol Neurodegener. 2019 Aug 29;14(1):36 Authors: Do J, McKinney C, Sharma P, Sidransky E Abstract Mutations in GBA1, the gene encoding the lysosomal enzyme glucocerebrosidase, are among the most common known genetic risk factors for the development of Parkinson disease and related synucleinopathies. A great deal is known about GBA1, as mutations in…
• For Gaucher Type 2, Prevail and Lonza Join to Develop Gene Therapy - Gaucher Disease News October 24, 2019 For Gaucher Type 2, Prevail and Lonza Join to Develop Gene Therapy  Gaucher Disease News
• Seelos Therapeutics Acquires Rights for Parkinson’s Gene Therapy Program July 8, 2019 Seelos Therapeutics has acquired the rights for a gene therapy program targeting the regulation of the SNCA gene, which provides instructions to make alpha-synuclein, a key player in the development of Parkinson’s disease. The accumulation of abnormal (misfolded) alpha-synuclein protein can result in toxic aggregates that lead to the death of dopaminergic neurons. These toxic aggregates are the main component…