Industry News

FDA Approves Crysvita for Victims of Tumor-Induced Osteomalacia

By Judy Ya-Hsuan Lin A number of patients globally suffer from excessive fibroblast growth factor 23 (FGF23). This rare disease causes tumors and induces a low phosphate concentration in the body and further softens and weakens bones. FGF23 plays a crucial role in regulating phosphate levels for bone maintenance, energy production, and nerve function. Excessive FGF23 diseases include X-linked hypophosphatemia (XLH) and tumor-induced osteomalacia (TIO). XLH affects approximately 3,000 children and 12,000 adults, while TIO inflicts about 500~1000 people in the US. Of the 500~1000 afflicted, “approximately half of all individuals with TIO, surgical removal of the tumors is not possible, leaving these patients with no other treatment options,” according to Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx Pharmaceutical Inc. In other words, such patients are only suitable for non-invasive treatment. On June 18th, Crysvita (burosumab-twza), co-developed by Ultragenyx Pharmaceutical Inc. and Kyowa Kirin Co., Ltd., was approved...
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FDA Approves Crysvita for Victims of Tumor-Induced Osteomalacia
North America

With NASH Drug Rejected, Intercept CEO Lambasts FDA’s Evolving Guidelines

By Sangeeta Chakraborty, Ph.D. In a complete response letter, the FDA handed an outright rejection to Intercept's fatty liver drug, obeticholic acid (OCA), much to the ire of its top brass. Intercept Pharmaceuticals had been a long time investor in the development and commercialization of novel therapeutics for the treatment of progressive non-viral liver diseases. OCA is one of its potential flagship drugs touted as a breakthrough therapy for the treatment of fibrosis in patients with non-alcoholic steatohepatitis (NASH).  NASH, an advanced and severe form of non-alcoholic fatty liver disease (NAFLD) is characterized by scarring and fibrosis of liver tissue. NAFLD starts with a buildup of fat in the liver due to preexisting obesity, high sugar diet, physical inactivity, and diabetes. This eventually causes inflammation, damage and impairs liver function giving rise to NASH. Symptoms are not readily apparent until the late stages. Primary treatments include lifestyle changes and dietary...
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With NASH Drug Rejected, Intercept CEO Lambasts FDA’s Evolving Guidelines
BIO Asia-Taiwan Feature

The Most Effective, Efficient and Targeted Business Partnering Platform Available!

With interest building and with the biggest names in biotech already signed up, BIO Asia  - Taiwan 2020 Online + Live, July 22-26, is expecting to host thousands of partnering meetings through the online BIO One-on-One Partnering™ system. This is your best chance to connect with the widest range of quality bioindustry contacts in Asia and beyond. As an online event, the system is open for both scheduling and partnering 24 hours a day over the five days of the conference. There's no travel, no jetlag, just plenty of opportunities! Sign up now to participate in this world-renowned partnering platform, and start filling up your meeting calendar! Join Free Webinar Training Course Join our webinar and learn how to take full advantage of the great new features of the BIO One-on-One Partnering™ system, including: • Self-schedule meetings during 5 full days of partnering from July 22-26, 2020. • View your partnering calendar...
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The Most Effective, Efficient and Targeted Business Partnering Platform Available!
Latest News

Verve Therapeutic’s One Time Base Editing Treatment Cuts Down Cholesterol Levels in Blood

By Ruchi Jhonsa, Ph.D. At the International Society for Stem Cell Research (ISSCR) 2020 virtual annual meeting, the “next-generation cardiovascular company” presented a potential one-time solution to the maladies of coronary heart disease. The rate of heart diseases has steadily surged, owing to sedentary lifestyles that have replaced physical activity and junk foods that have substituted healthy diets. Coronary artery disease is a common heart disease that takes millions of lives each year and is spiraling at a rapid rate. It is formed by the build-up of cholesterol and fatty deposit patches (plaques) that eventually blocks the arteries. Conventionally, coronary blockages are managed by blood thinners or cholesterol-reducing statins to keep blood pressure in check. However, health improvements are minimal due to poor adherence, high cost, drug-related side effects, and limited access to oral medications, especially in developing countries.   Base Editing to the Rescue Cambridge, MA-based Verve Therapeutics is...
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Verve Therapeutic’s One Time Base Editing Treatment Cuts Down Cholesterol Levels in Blood
Technology

Proximity RNA Sequencing: Breaking New Grounds in Spatial Transcriptomics

By Sangeeta Chakraborty, Ph.D. Background Displaying an extraordinary magnitude of compaction, the entire human genome of colossal dimensions (2 meters long) is squeezed right inside a micrometer-sized nucleus in a knot-free mass. Remarkably though, this genomic globule is accessible to a complex range of transcriptional machinery—through chromatin loops, which bring cis-acting DNA elements into proximity to transcriptional units—allowing a coordinated action of millions of molecules to drive the processes of the central dogma. Nuclear functions like transcription, recombination, replication, and several others rely on a well-compartmentalized genome and are constrained within their designated sub-nuclear territories rather than spread out randomly throughout the nucleus. Understanding these spatial territories and the interactions and networks of DNA and RNA they harbor, would be paramount to our understanding of how this massive nucleic acid architecture facilitates dynamic processes of gene regulation, cellular behavior, and development of the cell fate. Biological organization at this level...
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Proximity RNA Sequencing: Breaking New Grounds in Spatial Transcriptomics
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INTERVIEW

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Interview

Dr. Joseph Kvedar: The Potential of Telemedicine in the Era of Digitalization

Telemedicine is an intervention that facilitates patient access to healthcare outside of the doctor's office. To manage overwhelming patient populations, in recent years, hospital systems around the world have started to embrace it. The arrival of the COVID-19 pandemic has only accentuated the value of this approach with mandatory measures like lockdown and social distancing in place. "The interest in remote or virtual care delivery has been accelerating in the last five years, but our time to shine is now," says Dr. Joseph Kvedar, who has been a staunch advocate of digital health for close to three decades. A seasoned dermatologist at Harvard Medical School, Dr. Kvedar has been at the forefront of telemedicine with his eminent leadership at Partners...
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Dr. Joseph Kvedar: The Potential of Telemedicine in the Era of Digitalization
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INDUSTRY

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FDA Approves Crysvita for Victims of Tumor-Induced Osteomalacia

By Judy Ya-Hsuan Lin A number of patients globally suffer from excessive fibroblast growth factor 23 (FGF23). This rare disease causes tumors and induces a low phosphate concentration in the …

ASIA INSIGHT

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Keytruda Wins Big in China, Now Approved for Esophageal Squamous Cell Carcinomas

By Sangeeta Chakraborty, Ph.D. China’s National Medical Products Administration (NMPA) approved Keytruda (pembrolizumab), Merck’s blockbuster anti-PD1 drug, as monotherapy on 22nd June 2020 for the treatment of patients with locally …

TECHNOLOGY

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Proximity RNA Sequencing: Breaking New Grounds in Spatial Transcriptomics

By Sangeeta Chakraborty, Ph.D. Background Displaying an extraordinary magnitude of compaction, the entire human genome of colossal dimensions (2 meters long) is squeezed right inside a micrometer-sized nucleus in a …

NORTH AMERICA

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With NASH Drug Rejected, Intercept CEO Lambasts FDA’s Evolving Guidelines

By Sangeeta Chakraborty, Ph.D. In a complete response letter, the FDA handed an outright rejection to Intercept’s fatty liver drug, obeticholic acid (OCA), much to the ire of its top …

TODAY IN HISTORY

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