Orphan disease

Michigan State University Investigator Receives $2.1m to Study Existing Treatments for Select Rare Diseases

Oct 31, 2019 | Brain Cancer, Liver Cancer, Organ Failure, Orphan disease, Rare Diseases

Michigan State University (MSU) College of Medicine investigator Bin Chen just was awarded $2.1 million by the National Institutes of Health (NIH) to scour vast databases of existing drugs in a quest to treat orphan diseases. The investigative team received an...

FDA Clears IND for ABO-202 Gene Therapy Candidate for Infantile Batten Disease

May 24, 2019 | Infantile Batten Disease, Orphan disease, Rare Diseases

The FDA’s positive decision clears Abeona Therapeutics’ Investigatory New Drug Application (IND) to start a Phase I/2 trial. The studies will be conducted at infantile Batten disease global centers of excellence which include: University of Rochester Medical Center ...

Pfizer to Buy Rare Disease Portfolio Pipeline for $810 Million

May 14, 2019 | M&A, Orphan disease, Pfizer, Rare Diseases

Pfizer will buy Therachon Holding, a privately held, clinical-stage biotechnology company focused on rare diseases, with assets in development for the treatment of achondroplasia and short bowel syndrome (SBS). The deal is worth up to $810 million. Key Terms $340...

Michigan State University Investigator Receives $2.1m to Study Existing Treatments for Select Rare Diseases

FDA Clears IND for ABO-202 Gene Therapy Candidate for Infantile Batten Disease

Pfizer to Buy Rare Disease Portfolio Pipeline for $810 Million

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