Dorsal root ganglion

Capsida Biotherapeutics to Present Preclinical Data for Parkinson's Disease Associated with GBA Mutations Showing High Levels of GCase Enzyme Supplementation Following IV Administration

Retrieved on: 
Freitag, Mai 10, 2024
Traffic barrier, Patient, Cell, PD, Parkinson's disease, Baltimore Convention Center, Liver, Dorsal root ganglion, Disease, DSM-IV codes, Gene, Neuron, Cerebrospinal fluid, Brain, Society, Annual general meeting, GBA, Potential

THOUSAND OAKS, Calif., May 10, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced new preclinical data that demonstrate the potential of its next-generation intravenously (IV) administered gene therapy candidate, CAP-003, to safely and effectively treat Parkinson's disease (PD) associated with GBA mutations (PD-GBA). Collectively, the findings strongly support the best-in-class profile of CAP-003 and Capsida's plans to advance the therapy into clinical trials in the first half of 2025.

Key Points: 
  • Mutations in GBA, the gene expressing the GCase enzyme, are the most common genetic risk factor for PD.
  • Other treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to impact the disease.
  • Capsida's wholly owned novel gene therapy offers the potential to supplement the GCase enzyme with a single IV infusion, enabling long-term disease modification and substantially slowing disease progression.
  • "Our novel wholly owned PD-GBA gene therapy demonstrates the potential to provide long-term significant decrease in disease progression safely with a single IV infusion," said Capsida CEO Peter Anastasiou.

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: 
Dienstag, 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.

CANbridge and UMass Chan Medical School Spinal Muscular Atrophy Gene Therapy (CAN203) Improves Lifespan and Motor Function in Mice When Administered via Intracerebroventricular Injection

Retrieved on: 
Donnerstag, Mai 18, 2023
Neurology, Biotechnology, Pharmaceutical, General Health, Health, University, Genetics, Education, Tissue, SMN, ICV, SMA, Mouse, Central nervous system, University of Massachusetts, Gene, Spinal cord, Survival, SMN1, Toxicity, Research, Doctor of Philosophy, AAV, CNS, Brain cell, Patient, Dorsal root ganglion, Cell, DRG, Intracerebroventricular injection, American Society of Gene and Cell Therapy, IND, Society, Microbiology, Vaccine

The benchmark vector is identical in design to the only approved gene therapy for SMA.

Key Points: 
  • The benchmark vector is identical in design to the only approved gene therapy for SMA.
  • Furthermore, the second-generation gene therapy resulted in higher levels of SMN protein in the central nervous system (CNS), particularly within motor neurons, and lower expression in the peripheral tissues, compared to benchmark vector-treated mice.
  • Superior transgene expression was observed in disease-vulnerable cells, including ChAT-positive motor neurons in the spinal cord.
  • No evidence of DRG toxicity associated with superior SMN expression was observed at 90 days after ICV injection with the high dose of the second-generation gene therapy.

4D Molecular Therapeutics Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials & Development Plans for Fabry Disease Cardiomyopathy at WORLDSymposium™

Retrieved on: 
Mittwoch, Februar 22, 2023
Death, CPET, Safety, Quality of life, KCCQ, Heart, Real-time, ERT, Rituximab, FDA, Patient, Toxicity, GLS, Inflammation, Chelsea Handler, AAV, Biopsy, Dorsal root ganglion, RNA, Pharmaceutical industry, Medical device, Nursing

All three patients with 12 months of follow-up demonstrated improvement on cardiac contractility, exercise capacity and quality of life endpoints.

Key Points: 
  • All three patients with 12 months of follow-up demonstrated improvement on cardiac contractility, exercise capacity and quality of life endpoints.
  • Treatment was generally well tolerated, with transient acute aHUS being the only significant adverse event.
  • Data will also be presented at the WORLDSymposium™ 2023 in Orlando, Florida on February 25th, 2023.
  • We are developing 4D-310 for the treatment of Fabry disease cardiomyopathy, which is the primary cause of death and not addressed by current therapies.

ASLAN Pharmaceuticals Presents Late-Breaking Poster on Eblasakimab and Neuronal Itch Mechanisms at the 2022 Society for Investigative Dermatology Annual Meeting

Retrieved on: 
Freitag, Mai 20, 2022
Cannabinoid receptor type 2, Company, Disease, Dorsal root ganglion, U.S. Securities and Exchange Commission, Clinical and Translational Science, Nasdaq, Patient, SID, Quality of life, DHODH, Review, Cytokine, Clinical trial, Commission, COVID-19, AD, ASLN, Neuron, Projection, Autoimmune disease, Safety, Pathology, Society, LinkedIn, The Company, GLOBE, Atopic dermatitis, Pharmaceutical industry, Fine chemical

Chronic itch is a hallmark and major symptom of atopic dermatitis and other tType 2-driven inflammatory skin disorders.

Key Points: 
  • Chronic itch is a hallmark and major symptom of atopic dermatitis and other tType 2-driven inflammatory skin disorders.
  • In January 2022, ASLAN initiated the TREK-AD Phase 2b trial to evaluate the safety and efficacy of eblasakimab in moderate-to-severe AD patients.
  • ASLAN Pharmaceuticals (Nasdaq: ASLN) is a clinical-stage, immunology-focused biopharmaceutical company developing innovative treatments to transform the lives of patients.
  • These statements are based on the current beliefs and expectations of the management of ASLAN Pharmaceuticals Limited and/or its affiliates (the "Company").