Orphan Disease Articles & Analysis
31 news found
We believe that by using the same cells that promote tissue repair, organ maintenance, and immune system function, we can develop safe and effective therapies for some of the most difficult diseases and conditions. In 2024, we are focusing our efforts on two of our most promising programs: Hypoplastic Left Heart Syndrome (HLHS); and Alzheimer's Disease. ...
Application of induced pluripotent technology allows to fabricate tissues in diseased states, e.g., hereditary cardiac diseases, cardiac fibrosis, cancer and orphan diseases. ...
“With Teva’s strong foundation in neuroscience and our in-house expertise in neurology and psychiatry, this licensing and collaboration agreement adds a promising new compound to our early-stage pipeline as a possible orphan disease treatment for the growing patient population living with multiple system atrophy, as well as a potential option for ...
Commercial and Pipeline Update Product portfolio Acute Care Hospital: RYANODEX®, vasopressin, Barhemsys®, Byfavo® Oncology: BENDEKA, BELRAPZO, PEMFEXY11, TREAKISYM Japan12 75-person commercial team covers all products excluding BENDEKA and TREAKISYM Company projects growth in earnings while still supporting R&D Cash flow from legacy products expected to continue to ...
LB-P8 is, to the best of company's knowledge, the first drug to be granted orphan drug designation in the field of microbiome-based treatment for metabolic diseases. ...
At present, no PD-L1/4-1BB bispecific antibody has been approved for the treatment of pancreatic cancer worldwide. Orphan Drugs, also known as Rare Disease Drugs, refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. Orphan Drug Designations by the U.S. FDA are ...
It is being used to target multiple fibrotic diseases, including Idiopathic Pulmonary Fibrosis (IPF), and Hermansky-Pudlak Syndrome, a rare ‘orphan disease’ with no known treatment. ...
Epidermolysis bullosa, or EB, is a group of rare genetic skin diseases characterized by fragile skin that can lead to extensive blistering and wounding. It affects skin and mucous membranes, particularly of the gastrointestinal tract, genitourinary and respiratory systems. It is a debilitating disease affecting a small number of people, thus earning it an ...
ET 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 its financial results for the first quarter ended March 31, 2022 and provided a corporate update. ...
The company is also engaged in the discovery and development of innovative treatments and therapies for certain rare and orphan diseases as well as those affecting larger populations, such as diverticulosis. ...
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 ...
ET- 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 its financial results for the fourth quarter and full year 2021 and provided a corporate update. ...
He has spent most of his career in the hospital, orphan disease and oncology areas of the pharmaceutical and biotech industry cofounding Vidara Therapeutics and working for companies such as Pharmacia, Dendreon and Horizon Therapeutics. ...
OCU400 was granted four orphan drug disease designations from the FDA for treating four different gene mutation-associated retinal degenerative diseases between 2019 and 2020. The European Medicines Agency (EMA) granted Ocugen broad orphan medicinal product designation in 2021 for OCU400 for the treatment of both retinitis ...
INOVIO (NASDAQ: INO), a biotechnology company focused on bringing to market precisely designed DNA medicines to treat and protect people from infectious diseases, cancer, and HPV-associated diseases, today announced that several volunteers have been dosed with its DNA-based, intradermal Ebola vaccine candidate, INO-4201, as part of a randomized, ...
(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. ...
Data from the Phase 1 dose escalation phase of the study are expected in the first half of 2022 with top line clinical data from the Phase 2a expansion cohorts expected in the second half of 2022. About Orphan Drug Designation The term orphan drug refers to pharmaceutical products developed for the prevention, diagnosis and treatment of rare ...
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 ...
Lung Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel therapeutics for orphan, pulmonary diseases and conditions, today announced the appointment of Sharon Morriss, PhD, as Senior Vice President of Clinical Development. ...
Lung Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel therapeutics for orphan, pulmonary diseases and conditions, today announced the appointment of Cory M. ...