Posts Tagged Familial hypertrophic cardiomyopathy
Posted by Darshana V. Nadkarni, Ph.D. in Big Data -Cloud -IoT-Software -Mobile -Entrepreneurship, Biotech - Medical Device - Life Science - Healthcare on October 7, 2014
The Chinese Bioscience Association (CBA) is a non-profit organization, with a mission to promote education, networking, and community building for life science professionals.
At its recent annual conference in San Francisco Bay Area, an impressive array of speakers discussed new trends, promises, and challenges in the biotech industry.
In his keynote address, Dr. Frank McCormick, Professor at UCSF and founder of Onyx Pharmaceuticals, talked about targeted cancer therapies and some of the lessons learned from Onyx. Almost 30% of all mutated cancers happen in oncoproteins like BCR-ABL and RAS. In 1994, Onyx initiated a 5 year collaboration with Bayer that led to $25M study for drug development, based on RAS pathway. Unfortunately, it did not lead to drug leads. In hindsight, “we realized that it was too early to jump into the pathway based on our feeble understanding of RAS pathway”, said McCormick. However, Onyx has had several successes, despite the fact that finding new targets has become increasingly difficult, said McCormick. Sorafenib, a kinase inhibitor, was approved for treatment of renal and liver cancers. Onyx also has had success in viral therapies. In 2009, Onyx sued Bayer, its longtime partner, and won the lawsuit. Removal of the “change of control” clause in the original collaboration agreement, in favor of Onyx, has resulted in Onyx going from a $10M company in 1994 to $10B company in 2014.
Dr. Stephen Issacs, Chairman and Founder of Aduro Biotech talked about the importance of embracing change to keep growing. Issacs shared his story from 1978 where his career began in psoralen photochemistry and then teaching at UC Berkeley. In 1991, he found Cerus Corporation, a biomedical products company commercializing the Intercept Blood Systems. He raised over $650M and negotiated a $200M partnership with Baxter International and took the company public. He then found Aduro BioTech. Here is my previous article on his company Aduro Biotech http://bit.ly/JqDJ3K that uses a bacterial vector, in the form of genetically modified listeria, to stimulate the immune system to fight cancers and infectious diseases. Issacs also talked about his volunteer work in Kasigau, Kenya through his family non-profit organization, a cause that is very dear to his heart and to which he is highly dedicated.
Dr. Chandler Robinson shared his incredible story of how he went from being an undergraduate researcher to becoming a CEO of Tactic Pharmaceuticals, a privately held biotech, in Chicago. Tactic acquires and develops pre-clinical and clinical stage compounds. Somewhere in between his strenuous MD program at Stanford and pursuing his MBA in UK, Robinson acquired an abandoned drug, Decupratetm, and after couple of years, published his research in Science. In early 2013, Decupratetm got Orphan Drug Designation from European Commission for treatment of Wilson’s Disease. Wilson’s Disease is characterized by its disturbed copper metabolism that leads to copper accumulation in the body, resulting in severe disability or death. Tactic Pharma has acquired 3 compounds to date.
At Stanford Cardiovascular Institute (SVI), Dr. Joseph Wu and his team work with iPS cells (induced pluripotent stem cells). Wu is recipient of several awards and his clinical activities involve adult congenital heart disease and cardiovascular imaging. According to Wu, iPS will be a game changing platform for drug discovery research. Familial hypertrophic cardiomyopathy (HCM) is one of the most prevalent cardiovascular disease. At some point, we should be able take blood and make iPS cells, differentiate them into cardiac cells or other cell types, and expose them to different drugs to find out what would be the ideal drug for that patient, said Wu. Wu said, it is important to study different ethnicities; different ethnic groups metabolize drugs differently. CVI has received $20M grant and Wu’s team is trying to create a bank of 1000 cell lines, for drug discovery research. (iPSCs) seem to have exciting therapeutic implications in regenerative medicine, particularly for myocardial infarction and possibly for neurodegenerative diseases, diabetes and other disorders. However, stem cell biology is as yet incompletely understood and some of the challenges include immunogenicity, tumorigenicity and so on. Drugs can be tested on surrogates of patients, before giving them to the patients, said Wu.
Dr. Moninder Hora, SVP at Nektar Therapeutics talked about Nektar’s pipeline and technologies. Nektar’s technology is based on polymer conjugation platform, said Hora. Nektar has a robust pipeline of novel therapeutics and Nektar also partners with top biopharmaceutical companies to bring new products to market. Nektar’s pipeline of late stage molecules include Amaikacin, Ciprofloxacin, Fovista, BAX 858, and NKTR-102. Amaikacin Inhale is delivered directly to lungs to treat gram-negative ventricular pneumonia. NKTR-102 is first PEGylated small molecule, first in its class, to treat breast cancer. It has passed safety and efficacy markers and is showing to significantly improve overall survival and endpoints will be announced next year. Nektar is also getting positive results from some of its early stage molecules. Recently Nektar presented positive preclinical data for NKTR-214 for immunotherapy treatment. Nektar is developing pain management portfolio and recently got fast track designation for NKTR-181, a new oral opioid analgesic molecule for the treatment of moderate to severe chronic pain. NKTR has filed for regulatory approval in EU for MOVENTIG, comprising of once only daily oral tablet, to treat opioid induced constipation. Hora concluded saying that Nektar has very robust pipeline of highly promising molecules.
Dr. Zander Strange, VP of Bus Dev. and Corp. Dev. at Zosano Pharma shared about their products based on novel transdermal delivery technology. Zosano’s rapid onset system allows drug delivery to occur, almost painlessly. The drug is delivered close to the capillary bed, allowing it to quickly dissolve. The short-wear-time patch consists of microneedles coated with Zosano’s proprietary formulation of an existing drug, attached to an adhesive patch. Therapeutic effect is expected to occur within 30 minutes or less, in an easy, pain free administration. Zosano products are in dry formulation, thus vastly improving product stability and long shelf life, in addition to allowing for easy travel and storage at room temperature.
CBA annual conference was packed with impressive lineup of speakers, interspersed with plenty of networking opportunities. For additional information, please go to www.cbasf.org .
adult congenital heart disease, Aduro Biotech, Amaikacin, bacterial vector, BAX 858, Bayer, BCR-ABL, cardiovascular imaging, CBA, Cerus Corporation, Chandler Robinson, Chinese Bioscience Association Annual Conference, chronic pain, Ciprofloxacin, Decupratetm, diabetes, Dr. Frank McCormick, EU, European Commission, Familial hypertrophic cardiomyopathy, Fovista, HCM, http://bit.ly/JqDJ3K, immunogenicity, immunotherapy treatment, induced pluripotent stem cells, Intercept Blood Systems, iPS cells, Joseph Wu, kinase inhibitor, listeria, microneedles, Moninder Hora, MOVENTIG, myocardial infarction, Nektar Therapeutics, neurodegenerative diseases, NKTR-102, NKTR-181, NKTR-214, oncoproteins, Onyx Pharmaceuticals, opioid induced constipation, oral opioid analgesic molecule, PEGylated small molecule, photochemistry, product stability, Professor at UCSF, RAS, Regenerative Medicine, renal cancer, shelf life, Sorafenib, Stanford Cardiovascular Institute, stem cell biology, Stephen Issacs, SVI, Tactic Pharmaceuticals, targeted cancer therapies, transdermal delivery technology, tumorigenicity, UC Berkeley, viral therapies, Wilson’s Disease, www.cbasf.org http://bit.ly/1uwQ6af, www.eppicglobal.org, Zander Strange, Zosano Pharma
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