The science, research and development in cell and gene therapy that has originated from the Philadelphia region have resulted in four U.S. Food and Drug Administration approvals in just the past 21 months. These include: the first-FDA approved personalized cellular therapy for cancer (Kymriah™ by Novartis Pharamceuticals for advanced leukemia, August 2017); the first FDA-approved gene therapy (LUXTURNA™ by Spark Therapeutics for congenital blindness, December 2017); and a second approval of Kymriah™ by Novartis (for relapsed or refractory large B-cell lymphoma, May 2018).
Article by: PR Newswire; May 30, 2019
Philadelphia at the Forefront of Cell and Gene Therapy
Heritability and genome-wide association study of benign prostatic hyperplasia (BPH) in the eMERGE network
Benign prostatic hyperplasia (BPH) results in a significant public health burden due to the morbidity caused by the disease and many of the available remedies. As much as 70% of men over 70 will develop BPH. Few studies have been conducted to discover the genetic determinants of BPH risk. Understanding the biological basis for this condition may provide necessary insight for development of novel pharmaceutical therapies or risk prediction.
Article by: UroToday; April 28, 2019
2019 Distinguished Research Trainees Show Character and Creativity
Bumper scan of genomes pinpoints inherited risk factors for autism
Novel Gene Mutation found in Lymphatic Disorder
CAG researchers have identified a gene mutation that causes a serious lymphatic condition, and used that knowledge to restore normal lymphatic vessels in model animals. The laboratory findings may lead to a new therapy for patients with this type of abnormal lymphatic circulation.
MedicalXpress; August 28, 2018
Disease Susceptibility and Diagnostics
Using Edico Genome’s DRAGEN pipeline on 1,000 Amazon EC2 F1 instances, the Children’s Hospital of Philadelphia (CHOP) and Edico Genome set a new scientific world standard last week in rapidly processing whole human genomes. CHOP used the DRAGEN pipeline to process 1,000 whole pediatric genomes in two hours and twenty-five minutes.
Facebook-Hosted Panel Discussion; March 27, 2018
CHOP and Edico Set World Record For Genome Analysis
Using Edico Genome’s DRAGEN pipeline on 1,000 Amazon EC2 F1 instances, the Children’s Hospital of Philadelphia (CHOP) and Edico Genome set a new scientific world standard last week in rapidly processing whole human genomes. CHOP used the DRAGEN pipeline to process 1,000 whole pediatric genomes in two hours and twenty-five minutes.
Article by: Bio-IT World; October 23, 2017
Researchers identify gene variants linked to a high-risk children's cancer
Pediatric researchers in the Roberts Collaborative for Genetics and Individualized Medicine at Children's Hospital of Philadelphia have identified common gene variants that raise the risk of developing an aggressive form of the childhood cancer neuroblastoma. The discovery may assist doctors in better diagnosing subtypes of neuroblastoma.
Article by: EurekaAlert!; September 25, 2017
CHOP to lead new pediatric data resource center for research in childhood cancer, birth defects
The Center for Data Driven Discovery in Biomedicine at Children's Hospital of Philadelphia (CHOP) will lead a new, collaborative effort funded by the National Institutes of Health Common Fund to discover the causes of pediatric cancer and structural birth defects through the use of big data. The Center will be known as the "Kids First Pediatric Data Resource Center" (DRC).
Article by: EurekaAlert!; August 15, 2017
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