Award Data

Adeno-associated virus (AAV) vectors have been successfully employed in patients with rare neurological diseases. Recently, two AAV based gene therapy drugs have been approved by the FDA, Luxturna been valued at $850,000 with a one-time application for blindness and Zolgensma at $2,100,000 for spinal muscle atrophy. AAV vector mediated gene therapy has shown to be a potentially huge market. Althou ...

PROJECT SUMMARY Diabetic Retinopathy (DR) is a serious diabetic complication that is the leading cause of vision loss in working- age adults, affecting more than 7.5 million people in the USA. It causes more new cases of blindness than any other eye disease in people between the ages of 18 and 65. The anti-VEGF treatments can slow the progress of DR in many patients but are only used after signifi ...

PROJECT SUMMARY/ABSTRACT Obstructive sleep apnea (OSA) and obesity hypoventilation syndrome (OHS) are the most common types of sleep disordered breathing (SDB). OSA is highly prevalent in the United States, affecting 20-40% of the adult population and more than 50% of individuals with obesity. Approximately 20% of individuals with obesity develop OHS defined as daytime hypercapnia and hypoventilat ...

Project Summary Chronic pain is a prevalent, disabling problem affecting as many as 116 million adults in the United States. However, there are very few treatment options for chronic pain management, and those that are available are inadequate for the majority of those with this condition. Many also have significant negative side effects or are associated with significant adverse events. A growing ...

PROJECT SUMMARY Decades of modulating the anti-cancer activity of fluoropyrimidine drugs (FPs) thru schedule optimization, biochemical modulation, and drug combinations have provided a significant, but limited, survival advantage for treating colorectal cancer (CRC) patients with locally advanced or metastatic disease (mCRC). However, outcomes remain poor for patients with mCRC and since targeted ...

PROJECT SUMMARY/ABSTRACT CMTx Biotech is a drug development company working to commercialize a proprietary, clinical-stage, small- molecule drug candidate for the prevention of respiratory distress syndrome (RDS) and its long-term sequelae, bronchopulmonary dysplasia (BPD), in preterm infants. BPD is a chronic respiratory disease that occurs in preterm infants who develop RDS from a combination of ...

AbstractType 1 diabetes (T1D) is a chronic autoimmune disease in which insulin-secreting β-cells are destroyed by immune cells that infiltrate the pancreatic islets (i.e., insulitis). Instead of preventing or reversing T1D, most treatments focus on alleviating symptoms with insulin-replacement therapy. Meanwhile, T1D is a heterogeneous disease that poses significant challenges to define mechanism ...

PROJECT SUMMARYHigh blood pressure (BP), particularly high systolic BP (SBP), is the major modifiable risk factor for cardiovascular diseases (CVD) and related conditions. Indeed, over 50% of all adults have above-normal SBP (≥120 mmHg), placing them at increased disease risk. In addition, these adults face ~$2000/year in excess medical expenses compared to their peers with normal BP. Rates of B ...

ABSTRACT The goal of the proposed studies is to improve the efficiency of clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing in human pluripotent stem cells (PSCs), including embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs). Currently, CRISPR/Cas9 shows great potential for targeted gene editing, but remains challenging in PSCs due to their b ...

1 ABSTRACT2 Tuberous sclerosis (TS), also called tuberous sclerosis complex, is a rare, multi-systemic genetic and often life-3 threatening disease that causes benign tumors to grow in the brain and on other vital organs such as the kidneys,4 heart, eyes, lungs, and skin. Lymphangioleiomyomatosis (LAM) is a TS-related tumor-like disorder. Both occur5 as a consequence of an inherited or sporadic mu ...