Apic Bio, Inc., an innovative gene therapy company developing novel treatment options for patients with rare genetic diseases, U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for APB-102, the Company’s lead gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis (ALS) – a common cause of familial ALS. Source- Apic bio
ALS is a motor neuron disease (MND), is a debilitating, fatal, late-onset degenerative disorder of the nervous system characterized by motor neuron degeneration, which leads to progressive muscle wasting and weakness. The disease progresses rapidly; the median survival is three to five years, and only 20% of patients are alive five to ten years after symptom onset. Death usually results from respiratory failure. Despite the poor prognosis, there is considerable variation in the survival rate, and up to 10% of people with ALS live for more than eight years from the onset of first symptoms. The major breakthroughs in the etiology and pathophysiology of ALS are the discovery of various genetic risk factors such as mutations in SOD1, TARDBP, FUS, C9orf72, TBK1, and NEK1. Most of these risk factors were discovered only within the last ten years, and extensive research is underway to uncover the common mechanisms affected by these mutations that may lead to ALS, such as protein misfolding and aggregation.
Target patient pool; ALS prevalence in the United States ranges between 5.2 to 6.5 cases per 100,000 population where as incidence cases ranges between 0.6 to 2.8 cases per 100,000 population.
ALS market insight report comprehensively covers patient segmentation based on age, sex, mutation type (SOD1 mutations, TARDBP mutation, TDP-43, C9orf72 and others); onset sites according to handedness and footedness (divided into right and non-right dominant, Co-morbidities (coronary heart disease, arterial hypertension, myocardial infarctions, diabetes mellitus, hypercholesterolemia & Others), Symptoms (Dysphagia, Dyspnoea, Pain, Dysarthria, Cognitive changes, Sialorrhoea, Thickened saliva, Emotional lability, Depression and anxiety and Others), Amyotrophic lateral sclerosis (ALS) and its treatment in the 8 MM countries, covering the United States, EU5 (Germany, Spain, France, Italy, UK), Japan, and China
The current treatment market share, market uptake, attribute analysis with respect to the most potential emerging therapies (Rilutek, Radicava, Tiglutik, Arimoclomol, IONIS-SOD1Rx, Masitinib, NurOwn, Simdax, TW001, TW001, AMX0035, EPI-589, GM604, NP001, NSI-566, Reldesemtiv, RNS60, RNS60, etc.) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.
In terms of pharmacologic therapies, there are several pharmaceutical products are being approved as well as under different phases of development for the treatment. The key companies in to advance stage of developments are Orphazyme, Biogen, AB Science S.A, BrainStorm Cell Therapeutics, Tenax Therapeutics, Treeway biotechnology, Grifols, Amylyx Pharmaceuticals, Dainippon Sumitomo Pharma, Genervon Biopharmaceuticals, Neuraltus Pharmaceuticals, etc. targeting ALS. Looking at the current clinical development and assets progress card, fast development and robust product would bring a game-changing opportunity for the pharmaceutical company developing products around the treatment of ALS.
Thelansis is specialized in pharmaceutical market research and market Insight Report Company, published report across the therapeutic area which includes both rare / ultra-rare and mainstream indication. Over the period of time, we have built a strong repository of 6,000+ Bio-pharma reports which essentially covers Epidemiology study and Market forecasting based on the KOL opinions. Competitive intelligence and track of trial result throughout the phases of development executed by a team of a mix of Scientific and Business backgrounds. As an organization, the major focus is to provide real-world data evidence and market insight to pharmaceutical companies for their decision-making.
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