Hunter Syndrome Pipeline

DelveInsight’s Hunter Syndrome pipeline report provides comprehensive insights into 10+ companies and 10+ pipeline drugs in the Hunter Syndrome pipeline landscape. It also covers Hunter Syndrome pipeline drug profiles, including clinical and non-clinical stage products and therapeutics assessment by product type, stage, route of administration, and molecule type.

Some of the Important Findings from the Hunter Syndrome Pipeline Report

• There are currently 10+ drugs in the Hunter Syndrome pipeline.

• The Hunter Syndrome pipeline comprises therapies that are in different stages of the clinical phase including JR-141, RGX-121, DNL310, AVR-RD-05, and others, and are expected to launch in the upcoming years.

• Key pharmaceutical companies that are evaluating their drug candidates for Hunter Syndrome include JCR Pharmaceuticals Co., Ltd., Regenxbio Inc., Denali Therapeutics Inc, AVRO Bio, and others.

• Denali Therapeutics announced in March 2021 that the U.S. Food and Drug Administration (FDA) had granted Fast Track designation to DNL310 for the treatment of Hunter Syndrome patients (MPS II).

• The US FDA had granted Rare Pediatric Disease Designation to REGENXBI’s RGX-121 in August 2016.

• JR-141 is being developed and investigated in the Phase III studies by JCR Pharmaceuticals Co., Ltd.

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Hunter Syndrome: Overview

Hunter Syndrome, also known as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder characterized by the accumulation of large sugar molecules known as glycosaminoglycans (or GAGs or mucopolysaccharides) in body tissues. It’s a type of lysosomal storage disorder. Hunter syndrome is caused by a lysosomal enzyme deficiency called iduronate-2-sulfatase (I2S).

Hunter Syndrome Symptoms

The Hunter Syndrome symptoms range from mild to severe. The signs and symptoms include:

• An enlarged head

• Thickening of the lips

• A broad nose and flared nostrils

• A protruding tongue

Hunter Syndrome Treatment

Elaprase is the first and only approved treatment for Hunter syndrome. It is intended to replace the natural enzyme, increasing the catabolism of certain accumulated glycosaminoglycans (GAG), which abnormally accumulate in multiple tissue types in Hunter syndrome patients.

Hunter Syndrome Pipeline Analysis: Drug Profiles

DNL310: Denali Therapeutics Inc.

Product Description 

DNL310 (or ETV:IDS) is a recombinant IDS enzyme that has been engineered to cross the blood-brain barrier, replace the IDS enzyme, and treat the disease’s neuropathic and systemic forms.

Phase I/II

NCT04251026: Denali Therapeutics Inc. began a multicenter, multiregional, open-label study in July 2020 to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL310, an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT) designed to treat both peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II).

RGX-121: Regenxbio Inc.

Product Description 

RGX-121 is intended to use the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS).

Phase I/II

NCT04571970: Regenxbio Inc. Incorporated began a phase I/II study in November 2020 to check whether RGX-121 is safe, well-tolerated, and potentially effective in children aged five and up with severe MPS II.

Hunter Syndrome Pipeline Therapie and Key Companies 

• JR-141: JCR Pharmaceuticals Co., Ltd.

• RGX-121: Regenxbio Inc.

• DNL310: Denali Therapeutics Inc

• AVR-RD-05: AVRO Bio

For more information on the emerging drugs and companies, visit Hunter Syndrome Gene Therapy

Hunter Syndrome Therapeutics Assessment

• By Product Type

• Monotherapy

• Combination Therapy

• By Stage

• Discovery

• Pre-Clinical

• Phase I

• Phase II

• Phase III

• Pre-registration

• By Route of Administration

• Inhalation

• Intravenous

• Oral

• Subcutaneous

• By Molecule Type

• Small Molecule

• Stem Cell Therapy

• Gene Therapy

• Targets

• Immune System

• Multiple Kinase

• By Mechanism of Action

• Protease Inhibitors

• Immunomodulatory

Scope of the Report

Coverage: Global

Hunter Syndrome Key Players: JCR Pharmaceuticals Co., Ltd., Regenxbio Inc., Denali Therapeutics Inc, AVRO Bio, among others

Hunter Syndrome Pipeline Therapies: JR-141, RGX-121, DNL310, AVR-RD-05, and others

Table of Contents

For rich insights into Healthcare and Pharmaceutical News, visit Pharma, Healthcare, and Biotech Blog Posts

Key questions answered in the Hunter Syndrome Pipeline Report

• What Hunter Syndrome treatments are available?

• How many pharmaceutical companies are working on Hunter Syndrome treatments?

• Which of these companies’ pharmaceuticals is the most commonly used?

• How many Hunter Syndrome medications are manufactured by each company?

• How many Hunter Syndrome drugs are in the early, mid, or late stages of development?

• How many of the therapies that are currently being developed can be used alone or in combination with other treatments?

• What are the most significant industry-industry and industry-academy collaborations, mergers and acquisitions, and licensing practices that have an impact on Hunter Syndrome?

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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance.

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