orphan disease News
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Capricor Therapeutics to Participate in the Cantor Fitzgerald Rare Orphan Disease Summit
Capricor Therapeutics (NASDAQ: CAPR) a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that Dr. Linda Marbán, CEO of Capricor, will participate in a panel discussion at the virtual Cantor Fitzgerald Rare Orphan Disease Summit on March 30, 2022. Please see ...
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LISCure`s Phase 2 Drug Candidate Granted Orphan Drug Designation (ODD) from US FDA
LB-P8, a novel drug being developed by LISCure Biosciences, Inc. (LISCure) as a treatment for Primary Sclerosing Cholangitis (PSC), has been designated as an orphan drug by the US FDA. On December 1, 2022, the US FDA notified LISCure of the orphan drug designation of LB-P8, LISCure's microbiome novel drug, as treatment of PSC. LB-P8 is, to the best of company's knowledge, the first drug to be ...
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CCHS Foundation Announces Fifth International CCHS Day with Celebrity Champions Henry Winkler and Jonah Hill
The CCHS Foundation proudly announces the 5th Annual International CCHS Day on November 9, 2019. The CCHS Foundation is the fundraising arm of the CCHS Network, a support and advocacy organization for families living with Congenital Central Hypoventilation Syndrome (CCHS). CCHS is a multisystem disorder of the central nervous system where, most dramatically, the automatic control of breathing ...
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NuMedii Announces Strategic Research Collaboration with Two Leading Academic Research Institutions Utilizing Single-Cell Sequencing to Identify Precision Therapeutics for IPF
SAN MATEO, Calif., July 10, 2018 – NuMedii, Inc., today announced the formation of a strategic research collaboration with Yale School of Medicine and Brigham and Women’s Hospital with the goal of utilizing single-cell sequencing to identify novel precision therapies and biomarkers in idiopathic pulmonary fibrosis (IPF). An orphan disease, IPF is a chronic, progressive and usually ...
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NuMedii Announces Creation of World’s First Single-Cell Sequencing Atlas for Idiopathic Pulmonary Fibrosis by its Collaborators
SAN MATEO, Calif., July 9, 2020 /PRNewswire/ — NuMedii, Inc., a data-driven drug discovery company, today announced the availability of the world’s first single-cell sequencing atlas for idiopathic pulmonary fibrosis (IPF) that was the result of a strategic research collaboration including the Yale School of Medicine, Baylor College of Medicine, Brigham and Women’s Hospital, and ...
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Imaxio sells Trolovol® (D-penicillamine) to focus on vaccines
Imaxio, a biotech company specializing in immunology announces today the sale of Trolovol® (D-penicillamine), a drug for human usage indicated for an orphan disease affecting around 1,200 patients in France. The transaction was facilitated by CMC Consulting Group. This sale is aligned with Imaxio’s willingness to refocus its business on vaccines. This is the result of several years of ...
By IMAXIO S.A.
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AB2 Bio and WuXi Biologics Announce Collaboration to Accelerate Commercial-Scale Manufacturing of Tadekinig alfa
Tadekinig alfa is a novel IL-18 binding protein in pivotal Phase 3 testing for orphan disease IL-18 driven monogenic Hemophagocytic Lymphohistiocytosis (HLH) WuXi Biologics to undertake commercial scale manufacturing to support U.S. market launch AB2 Bio Ltd., a Swiss advanced clinical-stage biotech company developing innovative therapies for the treatment of severe systemic ...
By AB2 Bio Ltd.
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AB2 Bio expands Phase 3 trial of Tadekinig alfa in monogenic HLH
AB2 Bio Ltd, a Swiss advanced clinical-stage biotech company, specialized in developing innovative therapies for the treatment of severe systemic autoinflammatory diseases including rare diseases with high unmet medical needs, announces the expansion of its Phase 3 trial of Tadekinig alfa in the orphan disease IL-18 driven monogenic Hemophagocytic Lymphohistiocytosis (HLH). Recruitment to the ...
By AB2 Bio Ltd.
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LISCure Biosciences announces research collaboration with Mayo Clinic for rare liver diseases
LISCure Biosciences Inc., a leading biotech focused on microbial-based therapeutics, announced that it has executed a research collaboration agreement with Mayo Clinic for new drug development for rare liver diseases. LISCure has been conducting joint research with Mayo Clinic for NASH drugs since 2021, and this new collaboration is an agreement signed under the leadership of Nicholas F. ...
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Life Sciences Supply Chain Disruptions Likely to Persist through 2022
Supply chain disruption will be with biopharmaceutical companies for the foreseeable future, according to the recent CFO survey, Supply Chain Resilience, by BDO. A separate report by Catalent, Allogeneic and Autologous Cell Therapies Report 2022, agrees, saying, “Bottlenecks are one of the most common hurdles facing cell and gene therapy developers.” Drilling down to identify ...
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NuMedii Announces Research Collaboration With Leading Academic Research Institution to Explore New Targets and Therapeutics for Pancreatitis
SAN MATEO, Calif., November 1, 2018 – NuMedii, Inc., today announced the formation of a research collaboration with Johns Hopkins School of Medicine with the goal of discovering new targets and therapeutic options for pancreatitis. The collaboration brings together deep clinical expertise with NuMedii’s Artificial Intelligence Drug Discovery (AIDD) technology, which will enable the ...
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TVAX Biomedical Names Wayne Carter as President and CEO
TVAX Biomedical, a clinical stage development company advancing its proprietary T cell-based immunotherapy for the treatment of cancer, today announced the decision of the board to hire Dr. Wayne Carter as president and chief executive officer effective immediately. TVAX Biomedical is planning phase 2 clinical studies for glioblastoma multiforme, an orphan disease for which there is no effective ...
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Emmaus Life Sciences Announces Launch of Full-Service Telehealth Solution
Emmaus Life Sciences, Inc. (OTQXC: EMMA), a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, today announced the launch of an innovative full-service telehealth solution (https://www.endarirx.com/ask-physician) with its strategic partners, including Asembia LLC, US Bioservices Corporation and UpScriptHealth. The telehealth program capitalizes on the ...
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AVM Biotechnology Expands C-Suite in Anticipation of 2023 Commercialization Efforts
AVM Biotechnology, a clinical stage company developing AVM0703, a small molecule that mobilizes endogenous gamma delta TCR+ and invariant TCR+ bispecific Natural Killer T-like cells, announced the expansion of its senior management with the appointment of Brian Andersen as Chief Commercial Officer and Pearl Chan as Chief Financial Officer. These seasoned industry leaders will help drive the ...
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NuMedii Names Industry Veteran Heather Arnett, Ph.D. as Vice President of Research
SAN MATEO, Calif., November 1, 2018 – NuMedii, Inc., today announced the appointment of Heather Arnett, Ph.D., as Vice President, Research. Heather is leading research discovery and early development at NuMedii based on the company’s Artificial Intelligence Drug Discovery (AIDD) technology, which enables the discovery of novel targets and precision drug candidates across a number of ...
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Teva and MODAG Announce Licensing Collaboration for Neurodegenerative Disease Drug Candidate
TEL AVIV & WENDELSHEIM, Germany--(BUSINESS WIRE)-- Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) and MODAG GmbH today announced a strategic collaboration on the exclusive worldwide licensing and development of MODAG's lead compound anle138b and a related compound, sery433. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with ...
By MODAG GmbH
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Emmaus Life Sciences Presented Positive Real-World Data on the Efficacy of Endari in Preventing Acute Complications from Sickle Cell Disease at the 62nd Annual Scientific Meeting of the British Society for Haematology
Emmaus Life Sciences, Inc. (OTCQX: EMMA), a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, today announced real-world data on Endari®, the company’s prescription-grade L-glutamine oral powder, in preventing acute complications from sickle cell disease (SCD) and hemolysis in pediatric and adult patients in French Guiana and Qatar. The data ...
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Orphan Drug Designations by the U.S. FDA are meant to support the development of drug candidates that could potentially bring substantial therapeutic benefits
Antengene Corporation Limited (“Antengene” SEHK: 6996.HK), a leading innovative, commercial-stage global biopharmaceutical company dedicated to discovering, developing and commercializing first-in-class and/or best-in-class medicines for hematology and oncology, today announced that ATG-101, the company’s in-house developed novel PD-L1/4-1BB bispecific antibody, has been ...
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Emmaus Life Sciences Announces Partnership with UpScript to Provide Telehealth Solutions to Sickle Cell Disease Patients
Emmaus Life Sciences, Inc. (OTCQX: EMMA) a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease, today announced a partnership with UpScript IP Holdings, LLC. (UpScript), to offer telehealth solutions to sickle cell disease patients, expanding access to Endari, Emmaus’ prescription L-glutamine oral powder for the treatment of sickle cell disease. ...
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BIO Investor Forum Company Snapshot: InvivoSciences
BIO Investor Forum Company Snapshot: InvivoSciences What is your company’s lead product or technology? InvivoSciences, Inc. (IVS) is a leading developer of proprietary human 3D cell/tissue culture models for highly-predictive, multi-parameter phenotypic assays both in pharmacology safety & discovery screening and in personalized and regenerative medicine. IVS’s Functional 3D ...
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