IL2RG protein, encoded by tL2RG complementary DNA. (https://commons.wikimedia.org/wiki/File:Protein_IL2RG_PDB_2b5i.png) As reported in the 18 April issue of the New England Journal of Medicine, researchers at the St. Jude Children’s Research Hospital (Memphis, TN) and their colleagues have used gene therapy to restore immune function to eight infants with newly diagnosed X-linked severe combined immunodeficiency [...]
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Adeno-associated virus. Source: https://commons.wikimedia.org/wiki/File:Adeno-associated_virus_serotype_AAV2.jpg In recent weeks, buyouts of gene therapy companies by Big Pharmas or Big Biotechs—as well as other major gene therapy deals—have been making the news. Specifically, on February 25, 2019, leading gene therapy company Spark Therapeutics (Philadelphia, PA) announced that it had entered into a merger agreement with Roche. [...]
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Arming NK cells with enhanced antitumor activity. Source: Oberoi P, Wels WS - Oncoimmunology (2013) I attended and participated in an interactive breakout discussion session entitled “Targeting Solid Tumors with NK Cells” at the Cambridge Healthtech Institute conference “Discovery on Target” on Wednesday, September 26, 2018. The session moderator was Dan Kaufman, MD, [...]
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Checkpoint inhibitor therapies (NIH) On October 1, 2018, the The Nobel Assembly at the Karolinska Institute announced that it had awarded the 2018 Nobel Prize in Physiology or Medicine jointly to James P. Allison and Tasuku Honjo for their discovery of cancer immunotherapy via immune checkpoint inhibition. As is usual, these Nobel Prize [...]
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Lipid nanoparticle structure On August 10, 2018, Alnylam Pharmaceuticals (Cambridge, MA) announced the first-ever FDA approval of an RNAi (RNA interference) drug. The drug is Alnylam’s patisiran, which is indicated for the treatment of polyneuropathy due to transthyretin-mediated amyloidosis (ATTR). ATTR is a rare inherited, debilitating, and often fatal disease caused by mutations [...]
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Indoximod (1-methyl-D-tryptophan) In Chapter 2 of our 2017 book-length report, Cancer Immunotherapy: Building on Initial Successes to Improve Clinical Outcomes (published by Insight Pharma Reports), we discussed the major approved and emerging checkpoint inhibitors and checkpoint inhibitor modulators. Most of these, exemplified by the PD-1 inhibitors pembrolizumab (Merck’s Keytruda) and nivolumab (BMS’ Opdivo), [...]
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NIH Clinical Center On December 7, 2017 we published an article on this blog entitled ”Improving Candidate Selection: Translating Molecules into Medicines”. This article was based on a December 4, 2017 symposium sponsored by Aptuit entitled “Improving Candidate Selection: Translating Molecules into Medicines”. The focus of the meeting was on improving drug candidate [...]
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On January 12, 2018, Endpoints News sponsored a breakfast panel at the 2018 JP Morgan Healthcare Conference (JPM18) in San Francisco, CA. The focus of this panel was the current state of clinical cancer immunotherapy development. The full panel is recorded as a video on YouTube. The panel is also discussed in a special Web [...]
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Interface of retinal pigment epithelium and photoreceptor cells. Source: NIH Open-i As we discussed in our December 17, 2015 article on this blog, Spark Therapeutics’ (Philadelphia, PA) SPK-RPE65 had achieved positive Phase 3 results at that time. It was expected to reach the U.S. market in 2017. As announced by Spark in [...]
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Bromodomain. A chromatin "reader" that is a target of PPI drug development. Source: WillowW at the English language Wikipedia. Allan B. Haberman, Ph.D. was one of about 25 experts from pharmaceutical, biotechnology, and consulting firms who attended Aptuit’s one-day think-tank event, ”Improving Candidate Selection: Translating Molecules into Medicines”. This was the third [...]
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