How this biotech plans to go up against big pharma with its eye disease products
Shankar Musunuri has spent time in both the pharmaceutical world and the biotech arena – after 15 years at Pfizer (NYSE:PFE), he went on to launch several biotech start-ups. As co-founder, chairman &chief executive of Ocugen, he is looking to bring eye disease products to the patients that big pharma’s drugs have left out. “There’s so much unmet need when you look into retinal diseases,” Musunuri said. Get the full story at our sister site, Drug Delivery Business News. The post How this biotech plans to go up against big pharma with its eye disease products appeared first on MassDevice.
Asthma in Childhood
Stephen A. Tilles
STEPHEN A. TILLES, MD
IMMUNOLOGY AND ALLERGY CLINICS OF NORTH AMERICA
Not too many years ago many, practicing allergists viewed primary immunodeficiency disorders (PIDD) as a group of rare diseases with unknown underlying genetic defects, which were diagnosed based primarily on clinical presentation combined with rudimentary laboratory testing. With few exceptions, available treatments were unsatisfactory as well. In fact, despite realizing that real patients were suffering from these diseases, for many of us the world of PIDD was primarily of interest as a way of helping us understand how the immune system works.
Since the first genes associated with primary immunodeficiency were described in the early 1990s, there has been an exponential increase the number of genes found to have pathologic variants in patients with symptoms of primary immunodeficiency. Genetic testing currently used clinically includes chromosomal microarray, Sanger sequencing, and next-generation sequencing techniques, including whole exome testing. With the knowledge of the underlying molecular pathways, biologic therapies have been used for treatment and efforts are underway to broaden the availability of gene therapy.
The epidemiology of inflammatory bowel disease has changed over the past 4 decades. The incidence is rising dramatically and the age of onset has become younger. This changing landscape of inflammatory bowel disease reflects the new recognition that the youngest children with inflammatory bowel disease are enriched in cases with underlying primary immunodeficiency and monogenic causes. The management of these cases can be quite different, with specific genetic etiologies supporting unique interventions and some requiring hematopoietic cell transplantation for effective treatment.
This article focuses on loss of function STAT3 mutations causing autosomal-dominant hyper-IgE syndrome and dedicator of cytokinesis 8 deficiency, with discussion of other more recently described diseases.
Gastrointestinal (GI) involvement can be the presenting disease manifestation in patients with primary immunodeficiency disorders (PIDs). Infections and noninfectious diarrhea are frequent manifestations; however, malignancy and inflammatory and autoimmune-related GI diseases are also described. GI symptoms and disease seen in association with PIDs can mimic other diseases but are often resistant to conventional treatments owing to alternate disease mechanisms. Despite the advances in treatments for these conditions, therapy for immunodeficiency-related GI disease is often empiric.