Los Angeles, USA, Dec. 02, 2020 (GLOBE NEWSWIRE) -- Hunter Syndrome Clinical Trial Analysis of 10+ Companies Active in the Treatment Landscape
The Key companies developing drugs are Denali Therapeutics Inc., Shire, Regenxbio Inc., Green Cross Corporation, Takeda, Centogene AG Rostock, JCR Pharmaceuticals Co., Ltd., AVROBIO and others
DelveInsight’s, “Hunter Syndrome Pipeline Insight, 2020,” report proffers detailed insights about 10+ companies and 10+ pipeline drugs in Hunter Syndrome pipeline landscape. It includes pipeline drug profiles, covering the clinical, non-clinical stage products, and inactive pipeline products.
Key Takeaways of Hunter Syndrome Pipeline Report
- The companies and academics are working to evaluate the challenges and seek opportunities that could influence Hunter Syndrome R&D. The Hunter Syndrome therapies under development are zeroed in on novel approaches to treat Hunter Syndrome.
- JR-141 is currently in the phase III stage of development followed by TAK-609 and Hunterase in phase II clinical development and DNL310, RGX-121, SB-913 and TAK-531, AVR-RD-05 in phases I/II, I and IND clinical development.
- In October 2020, AVROBIO announced an exclusive, worldwide license agreement and a collaborative research funding agreement with The University of Manchester for an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, a rare and deadly lysosomal disorder that primarily affects young boys.
- Denali Therapeutics announces the publication of two new papers elaborating its blood-brain barrier delivery technology in science translational medicine in May 2020.
- The first Denali-authored paper by Kariolis et al. entitled “Brain delivery of therapeutic proteins using a novel Fc fragment blood-brain barrier transport vehicle in mice and monkeys,” published in STM, explains the development and engineering of the TV platform, and displays how it rises the brain exposure and distribution of specific antibodies in preclinical models including non-human primates.
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Hunter Syndrome aka Mucopolysaccharidosis type II (MPS II) is a condition, which affects many different parts of the body and occurs almost mainly in males. It is a progressively debilitating disorder.
Hunter Syndrome Emerging Drugs
- JR-141 is an anti-human transferrin receptor (J-Brain Cargo) antibody fused IDS (iduronate-2-sulfatase) that is anticipated to cross the blood-brain barrier (BBB). It comprises intact human IDS and anti-human transferrin receptor (hTfR) antibody that is being developed by JCR Pharmaceuticals. Presently, the drug is in phase III stage of clinical development for Hunter Syndrome patients in Japan and is in phase II preparation in Brazil. The company is also scheduling to commence another phase III trial for MPS II patients by March 2021.
- SHP-609/ TAK-609 is an investigational formulation of idursulfase administered intrathecally for the treatment of pediatric patients with Hunter syndrome and cognitive impairment. The drug is in phase II/III stage of clinical development for the patients with Hunter Syndrome and cognitive impairment, and the Shire is running the trial. However, Shire was acquired by Takeda in January 2019, and hopes to get the approval of TAK-609 for Hunter Syndrome by 2021.
- Hunterase by Green Cross eases the Hunter Syndrome symptoms. Hunterase was authorized by the Korean Food & Drug Administration (KFDA) in January 2012, for the treatment of Hunter Syndrome in Korea. It is presently in phase II stage of clinical development for the Hunter Syndrome patients in the United States. Moreover, GC Pharma has entered into a licensing agreement with Clinigen K.K. to commercialize Hunterase in Japan. In April 2020, Clinigen K.K. applied for the approval of Hunterase Intracerebroventricular (ICV) for the treatment of Hunter syndrome to Japan’s Medical and Medical Device Organization.
- DNL310 is a recombinant form of the iduronate 2-sulfatase (IDS) enzyme engineered to cross the BBB utilizing the proprietary Enzyme Transport Vehicle technology of Denali Therapeutics. DNL310 is intravenously administered and planned to improve overall clinical manifestations of Hunter syndrome. The company initiated a phase I/II stage clinical development of DNL310 for Hunter Syndrome patients in July 2020.
- RGX-121 is being developed as a novel, one-time for MPS II treatment, which is directly administered intra-cisternally into the Central Nervous System. It comprises of the NAV AAV9 vector encoding for human IDS (iduronate-2-sulfatase). Delivery of the enzyme, which is deficient within cells in the CNS could give a permanent source of secreted IDS beyond the blood-brain barrier, enabling the long-term cross-correction of cells throughout the CNS. This strategy could also proffer rapid IDS delivery to the brain. Currently, the drug is being investigated by Regenxbio in phase I/II stage of clinical development for paediatric patients with Hunter Syndrome.
- SB-913, a zinc finger nuclease (ZFN) in vivo genome editing product candidate to treat MPS II patients in development by Sangamo Therapeutics. It is an investigational product candidate that is designed to insert a normal copy of the IDS gene into a specific location in the DNA of liver cells. The aim of SB-913 treatment is to allow a patient's liver to create a continuous supply of functional IDS enzyme. Currently, the drug is in phase I/II stage of clinical development for patients with Hunter Syndrome.
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Key Questions regarding Current Hunter Syndrome Treatment Scenario and Emerging Therapies Answered in the report
- How many companies are developing Hunter Syndrome drugs?
- How many Hunter Syndrome drugs are being developed by the individual company?
- How many Hunter Syndrome emerging drugs are in mid-stage, and late-stage of development for the Hunter Syndrome treatment?
- What are the main collaborations (Industry–Industry, Industry-Academia), Mergers and acquisitions, licensing activities pertinent to the Hunter Syndrome therapeutics?
- What are the latest Hunter Syndrome treatment landscape trends, drug types and novel technologies developed to supersede the limitation of existing therapies?
- What are the clinical studies going on for Hunter Syndrome, and what is their status?
- What are the Hunter Syndrome key drug designations that have been granted to emerging drugs?
Scope of Hunter Syndrome Pipeline Therapeutics report
- Coverage: Global
- Major Players: 10+ key companies
- Key Drugs: 10+ products
- Phases:
- Hunter Syndrome Late-stage (Phase III)
- Mid-stage products (Phase II and Phase I/II)
- Early-stage products (Phase I/II and Phase I)
- Hunter Syndrome Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration:
- Subcutaneous
- Intravenous
- Intramuscular
- Molecule Type
- Molecule types:
- Recombinant fusion proteins
- Gene therapies
- Enzymes
- Product Type
- Product Types:
- Mono
- Combination
- Mono/Combination
Hunter Syndrome Emerging Drugs along with Key Players
- JR-141: JCR Pharmaceuticals
- TAK-609/SHP609/HGT-2310: Takeda
- GC1111 (Hunterase): Green Cross Corporation/GC Pharma
- DNL310: Denali Therapeutics
- RGX-121: Regenxbio
- SB-913: Sangamo Therapeutics
- AVR-RD-05: AVROBIO
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Browse Detailed TOC, Emerging Drugs and Key Companies @ https://www.delveinsight.com/report-store/hunter-syndrome-pipeline-insight
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