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Stoke Therapeutics, Inc. (NASDAQ: STOK) is a pioneering biotechnology company dedicated to addressing the root causes of severe genetic diseases. With a focus on developing antisense oligonucleotide (ASO) medicines, Stoke aims to increase gene expression by targeting RNA splicing. This innovative approach is designed to upregulate protein production, offering potential treatments for conditions that currently have limited therapeutic options.
One of the key technologies driving Stoke's mission is its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform. This advanced method allows for precise modulation of RNA to restore protein levels, providing a novel way to treat genetic disorders. The company's lead candidate, STK-001, is currently in clinical trials for the treatment of Dravet syndrome, a severe form of genetic epilepsy characterized by frequent and prolonged seizures. This promising candidate exemplifies Stoke's commitment to transforming the lives of patients with debilitating conditions.
Beyond Dravet syndrome, Stoke Therapeutics is actively exploring the application of its ASO platform to other severe genetic diseases. The company’s robust pipeline and strategic partnerships underscore its potential to deliver innovative therapies that address significant unmet medical needs.
Financially, Stoke Therapeutics has maintained a strong position, attracting strategic investments and partnering with leading research institutions to advance its clinical programs. The company primarily operates within the United States, leveraging its expertise and innovative technology to push the boundaries of genetic medicine.
For investors and stakeholders, Stoke Therapeutics represents a unique opportunity within the biotechnology sector, with its cutting-edge approach to genetic disease treatment and a clear focus on clinical and commercial milestones. Stay tuned for the latest updates and developments from Stoke Therapeutics as it continues to make significant strides in the field of genetic medicine.
Stoke Therapeutics (Nasdaq: STOK) has received authorization from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial (OSPREY) for STK-002, targeting autosomal dominant optic atrophy (ADOA), a rare inherited optic nerve disorder causing vision loss. The study aims to assess the safety and efficacy of STK-002, designed to upregulate OPA1 protein expression and potentially halt vision deterioration. Enrollment for the trial is expected to begin in early 2024. Currently, there are no approved treatments for ADOA, affecting approximately 1 in 30,000 people globally. Stoke's approach leverages the TANGO platform, focusing on treating haploinsufficiencies and CNS diseases.
Stoke Therapeutics (NASDAQ: STOK) announced that the FDA has approved a higher single dose of STK-001 (70mg) for the Phase 1/2a MONARCH study targeting children and adolescents with Dravet syndrome. This study, which aims to evaluate the safety and efficacy of STK-001, complements ongoing assessments in the ADMIRAL trial. Dravet syndrome is a severe genetic epilepsy affecting quality of life, with current treatments inadequate for most patients. Data from the new dose cohort is expected to be released in the second half of 2023, with the company aiming for a Phase 3 trial initiation in 2024.
Stoke Therapeutics (Nasdaq: STOK) announced progress in its STK-001 trials for Dravet syndrome, expecting to complete Phase 1/2a studies in 2023 and initiate Phase 3 in 2024. The company will deliver safety and efficacy data from various dosing cohorts throughout 2023. Financially, Stoke reported revenue of $12.4 million for 2022, a net loss of $101.1 million, and increased R&D expenses totaling $77.8 million. Stoke's cash reserves stand at $230.2 million, projected to fund operations until the end of 2025, supporting the advancement of STK-001 and STK-002.
Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology firm focused on treating severe diseases by enhancing protein expression through RNA-based therapies. The company will participate in two upcoming investor conferences: the Cowen 43rd Annual Health Care Conference on March 6, 2023, at 12:50 p.m. ET, and the 2nd Annual Needham Virtual Neuroscience Forum on March 15, 2023, at 3:20 p.m. ET. Live webcasts and replays will be accessible on Stoke's investor website. Stoke is known for its proprietary TANGO technology and is developing treatments for conditions like Dravet syndrome and autosomal dominant optic atrophy (ADOA). More details can be found on their website.
Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology firm focused on upregulating protein expression with RNA-based therapies, will present at the virtual SVB Securities Global Biopharma Conference on February 14, 2023, at 4:20 p.m. ET. This presentation will be in a fireside chat format and will be webcast live, available on Stoke's website for 30 days post-event. Stoke's lead compound, STK-001, targets Dravet syndrome, a severe genetic epilepsy, while STK-002 is under development for autosomal dominant optic atrophy. The company is headquartered in Bedford, Massachusetts, with operations in Cambridge, Massachusetts.
Stoke Therapeutics (NASDAQ: STOK) announced key milestones for 2023, focusing on its STK-001 therapy for Dravet syndrome. The company expects to complete Phase 1/2a studies this year to initiate a Phase 3 program in 2024. Additional safety data from the ongoing 45mg and 70mg dose cohorts is anticipated in mid and late 2023, respectively. As of September 30, 2022, Stoke had $252.2 million in cash, sufficient to fund operations until 2025. The firm is also advancing STK-002 for autosomal dominant optic atrophy (ADOA) towards clinical trials.
Nuvig Therapeutics, a biopharma company focused on autoimmune diseases, appointed Julie Anne Smith as CEO and Board member. With over 20 years in the life sciences sector, she has previously led biotech firms through drug development. Pamela Conley transitions to Chief Scientific Officer, having co-founded the company. Smith's leadership comes as Nuvig's lead program progresses towards IND-enabling activities. Nuvig aims to develop novel therapies to restore immune function without broad immunosuppression.
Stoke Therapeutics, Inc. (Nasdaq: STOK) will present at the 41st Annual J.P. Morgan Healthcare Conference on January 10, 2023, at 2:15 p.m. ET. The presentation will discuss the company’s innovative RNA-based medicines aimed at addressing severe diseases through upregulating protein expression. A live audio webcast will be accessible via the Investors & News section on Stoke’s website, with a replay available for 30 days post-event. Stoke is recognized for its proprietary TANGO approach and ongoing clinical trials for STK-001 in Dravet syndrome, as well as STK-002 for optic nerve disorders.