Dravet syndrome

Stoke Therapeutics Appoints Thomas Leggett as Chief Financial Officer

Retrieved on: 
Monday, April 29, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, Diamond, UBS, University, J.P. Morgan & Co., Dravet syndrome, Business administration, Columbia University, IPO, Bachelor of Economics, CPA, Patient, Initial public offering, MBA, Therapy, Economics, Growth

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 the appointment of Thomas (Tommy) Leggett as Chief Financial Officer.

Key Points: 
  • 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 the appointment of Thomas (Tommy) Leggett as Chief Financial Officer.
  • Mr. Leggett succeeds Stephen Tulipano, CPA, MBA, who resigned his position as Stoke’s Chief Financial Officer effective Tuesday, May 7.
  • Prior to joining Stoke, Mr. Leggett served as Chief Financial Officer at Affinia Therapeutics.
  • Before joining Affinia Therapeutics, Mr. Leggett was Chief Financial Officer at Black Diamond Therapeutics, where he led the Company’s $85 million Series C financing and $231 million IPO.

Encoded Therapeutics Provides Pipeline Updates From Its Vector Engineering Platform Ahead of Four Preclinical Presentations at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Tuesday, April 23, 2024
Biotechnology, Genetics, Health, Factorization of polynomials, Lennox–Gastaut syndrome, Brain, DRG, DSM-IV codes, SCN9A, Dorsal root ganglion, Dravet syndrome, ETF, Central nervous system disease, Gene silencing, LGS, Transgene, Neuroscience, Abstract, Code, CNS, GABAergic, Engineering, Res, Interneuron, MAPT, Angelman syndrome, Neuropathic pain, Central nervous system, Therapy, MicroRNA, Vaccine

Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.

Key Points: 
  • Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.
  • “Our ASGCT presentations showcase the depth and versatility of our vector engineering platform to develop highly specific precision therapies for CNS disorders with high unmet need,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded.
  • “Combining our novel regulatory elements and transgenes provides control of expression in target cells, potentially enabling treatment of devastating, intractable diseases.
  • Engineered transgenes include transcription factors (eTFs) that upregulate the expression of endogenous genes as well miRNA sequences derived from Encoded’s miRNA discovery platform.

Sangamo Therapeutics to Present Neurology-Focused Pre-Clinical Data From Its Epigenetic Regulation, Capsid Delivery and Genome Engineering Platforms at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Monday, April 22, 2024
Research, Infectious Diseases, Neurology, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, Science, STAC, Neuron, Central nervous system disease, Dravet syndrome, Central nervous system, MD, Sangamo Therapeutics, Cell, AAV, Tauopathy, CNS, Engineering, DNA, Charcot–Marie–Tooth disease, Patient, Gene, Society, DSM-IV codes, Prion, Angelman syndrome, ALS, Genome, Capsid, Therapy, Powder metallurgy

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.
  • Presentations will focus on the progression of Sangamo’s neurology-focused pre-clinical pipeline, including data supporting innovations in zinc finger epigenetic regulation, advances in AAV capsid engineering, and discovery of next-generation integrase technology.
  • These data will showcase a novel AAV capsid, STAC-BBB, that exhibited robust and widespread central nervous system transduction in cynomolgus macaques after intravenous delivery, as well as another novel AAV capsid, STAC-150, engineered to accelerate the discovery of potent and highly specific epigenetic regulators.
  • Sangamo will also present additional data from its AAV capsid engineering platform SIFTER and its efforts for integrase evolution and utilization.

Stoke Therapeutics Appoints Jason Hoitt as Chief Commercial Officer

Retrieved on: 
Wednesday, April 17, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Dravet syndrome, Gilead Sciences, College, Employment, Growth, Disease, Eteplirsen, Patient, Sarepta Therapeutics, Holy Cross, Marketing, Therapy, Autoimmunity, Vertex Pharmaceuticals, Pharmaceutical industry

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 the appointment of Jason Hoitt as Chief Commercial Officer.

Key Points: 
  • 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 the appointment of Jason Hoitt as Chief Commercial Officer.
  • As Chief Commercial Officer and a member of Stoke’s leadership team, Mr. Hoitt will be responsible for overseeing the Company’s global commercial strategy for STK-001.
  • "Jason brings deep expertise and a proven track record in successfully driving commercial strategies for novel new medicines and accelerating growth for companies as they transition to the commercial stage,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics.
  • Prior to Provention Bio, Mr. Hoitt served as Chief Commercial Officer at Dova Pharmaceuticals and led all commercial efforts including launch strategy and execution for DOPTELET® (avatrombopag) for chronic immune thrombocytopenia.

Avicanna Reports Full Year 2023 Audited Financial Results 

Retrieved on: 
Tuesday, April 2, 2024
Cream, Cannabidiol, Cannabinoid, Dravet syndrome, Brazilian Health Regulatory Agency, Interest, Medical cannabis, Seizure, Business, Lennox–Gastaut syndrome, Patient, Pharmacy, Active ingredient, Research, Acquisition, RDC, Ultra, Stock, CBD, Insurance, Health Canada, Bank statement, GMP, Dermatology, TSX, Therapy, Pharmaceutical industry

TORONTO, April 02, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce year end 2023 results and audited financial statements.

Key Points: 
  • TORONTO, April 02, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce year end 2023 results and audited financial statements.
  • Cash used in operations of $1.38 million; an 81% reduction compared to $7.4 million in 2022.
  • “2023 was a tremendous year for Avicanna where we solidified of our leadership position in the Canadian medical cannabis sector through our proprietary products and the launch of MyMedi.ca.
  • We have successfully demonstrated the scalability of our revenues and made significant improvements in our fundamentals while advancing all our business pillars.

Avicanna Announces the Canadian Launch of 10% CBD (THC Free) Proprietary Formulation

Retrieved on: 
Wednesday, March 27, 2024
Cannabinoid, Absorption, Dravet syndrome, Seizure, Lennox–Gastaut syndrome, Patient, Research, CBD, Health Canada, TSX, THC, Plasma protein binding, RHO

TORONTO, March 27, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce the launch of RHO Phyto Micro Drop 100, a 10% CBD (THC free), proprietary oral formulation in Canada. RHO Phyto Micro Drop 100, is designed to deliver enhanced absorption of cannabinoids through an inverted emulsion technology. RHO Phyto Micro Drop 100 will be available to Canadian patients with medical authorization exclusively at the MyMedi.ca medical cannabis care platform.

Key Points: 
  • TORONTO, March 27, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce the launch of RHO Phyto Micro Drop 100, a 10% CBD (THC free), proprietary oral formulation in Canada.
  • RHO Phyto Micro Drop 100, is designed to deliver enhanced absorption of cannabinoids through an inverted emulsion technology.
  • RHO Phyto Micro Drop 100 will be available to Canadian patients with medical authorization exclusively at the MyMedi.ca medical cannabis care platform.
  • RHO Phyto Micro Drop 100 contains the same proprietary oral formulation as the product Trunerox™.

Stoke Therapeutics Announces Landmark New Data That Support the Potential for STK-001 to be the First Disease-Modifying Medicine for the Treatment of Patients with Dravet Syndrome

Retrieved on: 
Monday, March 25, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, FDA, Genetics, Clinical Trials, Diagnosis, Neurology, Socialization, Communication, Disease, Seizure, Patient, Fenfluramine, University, OLE, Behavior, Cognition, Therapy, Natural history, FAES, Dravet syndrome, Movement, Pediatrics, Food, Medicine, Pharmaceutical industry

Data from these studies showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification.

Key Points: 
  • Data from these studies showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification.
  • These improvements were observed among a highly refractory group of patients who were already taking the best available anti-seizure medicines.
  • Today, the Company also announced clearance from the U.S. Food and Drug Administration (FDA) that allows patients to receive three doses of 70mg followed by continued dosing at 45mg.
  • “STK-001 is the first medicine in development to demonstrate substantial and durable reductions in seizure frequency and improvements in multiple measures of cognition and behavior.

Takeda Announces Positive Topline Results from Phase 2 Study Evaluating Mezagitamab (TAK-079), a Potential Best-in-Class Anti-CD38 Monoclonal Antibody, for Primary Immune Thrombocytopenia

Retrieved on: 
Wednesday, March 13, 2024
Neurology, Biotechnology, Pharmaceutical, Oncology, General Health, Health, FDA, Clinical Trials, Fast track (FDA), Psoriasis, Cell, Disease, Risk, Patient, Megakaryocyte, Immunoglobulin G, ITP, B cell, Quality of life, Platelet, Autoantibody, Gastrointestinal tract, CD38, Bleeding, Safety, Dravet syndrome, Blood, Lennox–Gastaut syndrome, Fatigue, Fiscal, Food, Autoimmune disease, Pharmaceutical industry

Takeda ( TSE:4502/NYSE:TAK ) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP).

Key Points: 
  • Takeda ( TSE:4502/NYSE:TAK ) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP).
  • An interim analysis of the ongoing Phase 2 study demonstrated positive safety and efficacy results.
  • Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024.
  • Results from the Phase 2 trial have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

Ovid Therapeutics Reports Business Updates, Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Friday, March 8, 2024
Graviton, Epilepsy, Pharmacy technician, CCM, Research, Transcranial magnetic stimulation, DS, Neuron, Vigabatrin, LGS, Psychosis, Biomarker, Element, Ligand Pharmaceuticals, Lennox–Gastaut syndrome, Patient, Acceleration, Medicine, Status epilepticus, Dravet syndrome, ROCK2, KCC2, Hemangioma, IND, Seizure, Pharmaceutical industry

In addition, Takeda is expected to read out results from two pivotal Phase 3 studies evaluating soticlestat.

Key Points: 
  • In addition, Takeda is expected to read out results from two pivotal Phase 3 studies evaluating soticlestat.
  • Of these potential future payments, Ovid sold a 13% interest to Ligand Pharmaceuticals for $30 million in October 2023.
  • Financial runway: Ovid anticipates its cash runway will support operations and clinical development programs into the first half of 2026.
  • Ovid also anticipates several events for its current pipeline programs and clinical results for soticlestat from Takeda in 2024.

Encoded Therapeutics Announces UK CTA Approval for Dravet Syndrome Gene Therapy Candidate ETX101

Retrieved on: 
Monday, February 26, 2024
Children, Neurology, Genetics, Clinical Trials, Health Technology, Biotechnology, General Health, Pharmaceutical, Consumer, Health, Royal Hospital, Central nervous system, Health care, Therapy, CNS, POLARIS, MHRA, Dravet syndrome, IND, AAV, CTA, Medication, Pharmaceutical industry

Dravet syndrome is the most common developmental and epileptic encephalopathy and is characterized by a spectrum of clinical symptoms, including treatment-resistant seizures and neurodevelopmental stagnation.

Key Points: 
  • Dravet syndrome is the most common developmental and epileptic encephalopathy and is characterized by a spectrum of clinical symptoms, including treatment-resistant seizures and neurodevelopmental stagnation.
  • The UK CTA for EXPEDITION is part of a global regulatory strategy established by Encoded for clinical development of ETX101, which also includes regulatory clearance for an investigational new drug (IND) application in the United States and approval under the Clinical Trial Approval (CTA) scheme in Australia.
  • “This CTA marks the third regulatory approval for ETX101 as we advance our global clinical development program, POLARIS, to bring this potential one-time AAV gene regulation therapy to people affected by SCN1A+ Dravet syndrome around the world,” said Sal Rico, M.D., Ph.D., Chief Medical Officer of Encoded.
  • EXPEDITION represents a significant stride forward in our collective pursuit of new treatments that can truly make a difference in the lives of people affected by Dravet syndrome."