Part A of the study will test two comparatively low doses of STK-001. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. More recently, Stoke announced that the FDA had given clearance to begin dosing in part A of the MONARCH clinical trial. Revive is a life sciences company focused on the research and development of therapeutics for infectious diseases and rare disorders, and it is prioritizing drug development efforts to take advantage of several regulatory incentives awarded by the FDA such as Orphan Drug, Fast Track, Breakthrough Therapy and Rare Pediatric Disease designations. Dravet Syndrome Clinical Research Trials | CenterWatch Stoke Therapeutics Inc Sr. Manager/Manager, Clinical ... Candidate. Stoke believes that STK-001, a proprietary antisense . Stoke Therapeutics : Presents Data from the Phase 1/2a ... Stoke Therapeutics believes STK-001 can be the first disease-modifying therapy that targets the underlying cause of Dravet syndrome. Rationale: DS is a severe and progressive genetic epilepsy that typically begins in the first year of life and is characterized by high seizure frequency and severity, intellectual disability, motor abnormalities, and a high risk of SUDEP.Most cases are caused by spontaneous, heterozygous loss of function mutations in the SCN1A gene, which encodes for voltage-gated sodium channel type 1α . Behind Stoke, gene therapies are approaching the clinic with Encoded Therapeutics having the most advanced clinical candidate and preparing for trials in 2021. The candidate will oversee execution of data management activities for assigned clinical trial (s) and contribute to overall clinical study data strategy and planning. The trial plans to enrol approximately 22 children and adolescents with Dravet syndrome across various clinical sites in the UK. bedford, mass., december 03, 2021 -- ( business wire )-- stoke therapeutics, inc. (nasdaq: stok), a biotechnology company dedicated to addressing the underlying cause of severe diseases by. Chicago, IL. Stoke is seeking an enthusiastic, motivated Clinical Data Manager. The drug is in Phase III clinical studies for the treatment of Dravet syndrome. 8:30 am Chair's Opening Remarks Jeremy Levin CEO & Chairman, Ovid Therapeutics Investigating Diverse Therapeutic Modalities to Treat Neurodevelopmental Disorders 8:40 am Panel Discussion - Diving into Specific Therapeutic…Read more A quick information sheet can be found here. Join to Connect . BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a . STK-001 is an investigational new medicine for the treatment of Dravet syndrome that is currently being evaluated in ongoing clinical trials. An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome. Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. In agreement, clinical trials are currently testing different classes of microbiome-based therapeutics for a range of conditions (Table 1). STK-001 is an anti-sense oligonucleotide (ASO). Biotech company Stoke Therapeutics is set to commence dosing in the ongoing Phase I/IIa MONARCH trial of STK-001 in children and adolescents with Dravet syndrome. Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely u . BEDFORD, Mass. Stoke Therapeutics(Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines. The latest move comes after the company received Investigational New Drug Program (IND) approval from the US Food and Drug Administration (FDA) in November 2020 to advance orismilast into a Phase IIb study. Stoke believes that STK-001, a proprietary antisense . November 8, 2021 - Company nominates STK-002 as clinical candidate for the treatment of autosomal dominant optic atrophy (ADOA), the most comm . The results will inform on the long-term safety of repeat administration of STK-001 and will help determine appropriate and effective STK-001 dosing in future clinical studies. About Stoke Therapeutics. Using the Company's . Explore 399,790 research studies in all 50 states and in 220 countries. The company now intends to test STK-001 in a Phase 1/2 clinical study in the first half of 2020. About STK-001 STK-001 is an investigational new medicine for the treatment of Dravet syndrome that is currently being evaluated in ongoing clinical trials. View this and more full-time & part-time jobs in Cambridge, MA on Snagajob. Clinical efficacy as well as safety profile of these delivery approaches will be covered. 781-303-8302. dkalmar@stoketherapeutics.com. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. "During the last year, the power and potential of . Detailed Description: About Stoke Therapeutics. "Stoke science is leading us toward solutions that we believe will make a real difference for patients and their families. In close collaboration with Clinical Operations Trial Lead (s), will ensure compliance with quality standards . The candidate will oversee execution of data management activities for assigned clinical trial(s) and contribute to overall clinical study . Pratik Patel Senior Clinical Operations Associate at Stoke Therapeutics Boston, Massachusetts, United States 500+ connections This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. the risk that positive results in a clinical trial . Stoke Therapeutics is evaluating the long-term safety & tolerability of repeated doses of STK-001 in patients with Dravet syndrome who previously participated in studies of STK-001. Posting id: 699563510. "The need for a medicine that will treat the underlying cause of Dravet syndrome is clear to . Ph.D. Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2a study (ADMIRAL) of STK . Stoke Therapeutics just IPO-ed in August last year, and someone was saying that the company- with one preclinical asset valued at $900mn market cap - was an easy short.Fast forward to December . Stoke Therapeutics is pioneering a new way to treat the underlying causes of severe #geneticdiseases by precisely upregulating protein expression. The drug showed that 70.6% of patients receiving the drug had a decrease in convulsive seizure frequency between Day 29 and Day 84. The Company expects to share preliminary clinical data on 30mg MAD doses of STK-001 in the second half of 2022. Clinical trial information can be found on ClinicalTrials.gov. - Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2a study . UNION therapeutics has enrolled the first participant in the IASOS Phase IIb study of oral orismilast in psoriasis patients. Clinical trials have already started, with Stoke Therapeutics initiating the first clinical trial with a disease-targeting therapy in Dravet syndrome in summer 2020. Stoke was launched in 2018 with a $40 million Series A investment funded by Apple Tree Partners. Corporate Presentation - November 2021. We are committed to working with a sense of urgency while we conduct robust clinical trials that are designed to provide the necessary evidence to gain approval." - Nancy M. Wyant, SVP, Development Operations University of Chicago. About Stoke Therapeutics. Detailed Description: About Stoke: Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines. Sep 2007 - Jul 20135 years 11 months. Stoke Therapeutics Inc (NASDAQ: STOK) announced highlights from five presentations related to the ongoing clinical development of STK-001 being made at the American Epilepsy Society 2021 Annual . Stoke is seeking an enthusiastic, motivated Clinical Data Manager. Research focused on drug repurposing in pre-clinical models of pulmonary fibrosis. Change in seizure frequency, overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study. The candidate will oversee execution of data management activities for assigned clinical trial (s) and contribute to overall clinical study data strategy and planning. The company expects initial data from the . Working closely with the clinical research staff in the MGH Cancer Center, the . Stoke believes that STK-001, a proprietary antisense . Stoke believes that STK-001, a . Published: Dec 06, 2021 By Alex Keown. Financial Third Quarter 2021 Financial Results. Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. STK-001: Stoke Therapeutics STK-001 is an antisense oligonucleotide that increases levels of SCN1a mRNA in the cell resulting in increased NaV1.1 protein production. Lyra Therapeutics (Nasdaq: LYRA) is a clinical-stage company pioneering a new therapeutic approach to treat debilitating ear, nose and throat (ENT) diseases that impact the lives of millions of . BEDFORD, Mass., December 3, 2021 - Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced highlights from five presentations related to the ongoing clinical development of STK-001 being made at . See listed clinical studies related to the coronavirus disease (COVID-19) ClinicalTrials.gov is a resource provided by the U.S. National Library of Medicine. For more detailed information, please visit this link or email [email protected]. Apply online instantly. Stoke Therapeutics presented data on the effectiveness of its STK-001 in Dravet syndrome. 08 Oct 2020. The FDA has lifted the clinical hold on Spero Therapeutics Inc's (NASDAQ: SPRO) Phase 2 trial of SPR720 for nontuberculous mycobacterial (NTM) disease. Stoke Therapeutics is now enrolling for the MONARCH study to assess if this works as a treatment for patients with Dravet syndrome. BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced highlights from five presentations related to the ongoing clinical development of STK-001 being made at the American Epilepsy Society (AES) 2021 Annual . Apply for a Stoke Therapeutics Inc Sr. Manager/Manager, Clinical Operations job in Cambridge, MA. Former Sarepta Therapeutics CEO Ed Kaye is planning a clinical trial, partnerships and an IPO for his new biotech startup, Stoke Therapeutics, after closing a $90 million Series B round this week. Stoke Therapeutics | 5,126 followers on LinkedIn. STK-001 is expected to be the first disease-modifying treatment to target the disease's genetic cause. About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines. Conclusion. The CTM is accountable for the day-to-day operations of . stoke therapeutics, inc.(nasdaq: stok), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with rna-based medicines, today announced that five abstracts related to the company's work in dravet syndrome will be presented at the american epilepsy society(aes) 2021 annual … Stoke aims to develop the first precision medicine platform to . In close collaboration with Clinical Operations Trial Lead (s), will ensure compliance with quality standards . Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study. Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of severe genetic diseases, including genetic conditions affecting the central nervous system, eye, and liver. Jan. 4, 2022, 11:38 AM. Posting id: 699563510. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the presenter or Stoke or any officer . Table 1 Examples of microbiome-based therapeutics in . The SPR720 program . Please refer to the brochure for other time zones. Senior Clinical Supply Chain Manager at Stoke Therapeutics Greater Boston 500+ connections. The FDA had previously placed higher doses of the therapy on a partial clinical hold, meaning they could not be tested in the trial. Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates. Trials Show Hope on the Horizon for Dravet Syndrome, Glioblastoma. In a clinical trial update, Nkarta, Inc. (NASDAQ: NKTX) said it has commenced the clinical trial evaluating NKX019 in CD19+ advanced B cell malignancies. For now, participation in a clinical trial is the only way to access our investigational medicines. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year. Day One Tuesday December 14, 2021 Day Two Wednesday December 15, 2021 All times shown in EST. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. Gene Therapy Clinical Study in Adult PKU. Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. This trial is evaluating STK-001 in children and adolescents, ages 2-18 years, with Dravet syndrome. The reconciliation of utilitarian and equitable approaches to pandemic triage through a universal ethical proposition for allocation of critical care health resources is ineffective in most of the developing world primarily due to their large population size/density, and limited pre-existing healthcare availability. Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates - Company nominates STK-002 as clinical candidate for the treatment of autosomal dominant optic atrophy . Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with RNA-based medicines, today reported financial results for the full year ended December 31, 2020 and provided business updates. to fund research, clinical and process . Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. STK-001 is part of a new technology called TANGO that Stoke Therapeutics is leveraging to treat haploinsufficiencies like Dravet syndrome. Target Audience and Goal Statement. Funding: Please list any funding that was received in support of this abstract. The goal of this activity is to increase clinicians' awareness of and ability to select an appropriate anti-epilepsy therapy for patients with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC), and to highlight the need to correctly diagnose patients with . Stoke Therapeutics Announces Presentations from the Company's Dravet Syndrome Program at the American Epilepsy Society 2021 Annual Meeting . Apply for a Stoke Therapeutics Inc Sr. Manager/Manager, Clinical Operations job in Cambridge, MA. STK-001 is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in a Phase 1/2a clinical trial. The Clinical Trial Manager I (CTM I) is responsible for the planning, implementation, and conduct of regional clinical trials in Phase I-IV. Change in seizure frequency, overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study. : Stoke Therapeutics. Net loss for the three months ended September . YEAR FOUNDED: Apply online instantly. Stoke Therapeutics USA Listed Stoke Therapeutics (Nasdaq: STOK), is a clinical-stage biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. . Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. stoke therapeutics, inc. (nasdaq: stok), a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating protein expression with rna-based medicines, today. Stoke Therapeutics, Inc.,, a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced the . Stoke Therapeutics to progress MONARCH trial for Dravet syndrome. A new type of medicine, STK-001, designed by Stoke Therapeutics increases healthy copies of the Nav1.1 sodium channel in pre-clinical animal and cell models. Chief Communications Officer. December 3, 2021 View this and more full-time & part-time jobs in Cambridge, MA on Snagajob. "During the first half of 2021, the Stoke team laid the groundwork for important clinical milestones in the second half of this year," said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. STK-001 is an investigational new medicine for the treatment of Dravet syndrome that is currently being evaluated in ongoing clinical trials. This required a deep . The goal of the session is to update on clinical progress, highlight the advantages of oligo therapeutics for these related organs, and discuss potential issues related to oligo delivery. Stoke believes that STK-001, a proprietary antisense . Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. Change in seizure frequency, overall clinical. Stoke does not offer programs that allow access to our investigational medicines prior to regulatory approval. An investigational drug for Dravet syndrome shows promise in treating the progressive form of genetic epilepsy. 31 Mar 2021 Stoke Therapeutics is set to commence a Phase I/IIa ADMIRAL study of its investigational new medicine, STK-001, for treating Dravet syndrome, a severe and progressive genetic epilepsy. 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