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Imagine a single dose of medicine priced at $3.5 MILLION. Since the approval of the gene therapy Hemgenix , this is a reality. How can this be fair? Stay tuned to better understand the costs and risks of drug development, learn about the world's most expensive gene therapy and small molecule, and learn some new things about science.

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00:00 Absurdly expensive drugs what you will learn today
01:20 Honorary mentions (Skysona, Zynteglo, Soliris)
02:57 Pharma business basics: R&D cost, peak sales, functional cost, grosstonet, probability of success
05:13 Most expensive small molecule: An "ultraultra rare disease drug" (lonafarnib, Zokinvy)
08:45 Organic chemistry and process synthesis
11:05 Most expensive gene therapy: Hemgenix, hemophilia B, how gene therapies work
14:20 The best way to learn: Brilliant.org
15:32 How many sales Hemgenix might get Explained
18:49 Recap and closing remarks

This video was sponsored by Brilliant.

Disclaimer This channel does not provide medical advice!
No information on this channel is intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding a medical condition or treatment and before undertaking a new health care regimen, and never disregard professional medical advice or delay in seeking it because of information on Youtube.

Key references:
Lonafarnib: First Approval | Drugs. 2021; 81(2): 283
A Novel IodideCatalyzed Reduction of Nitroarenes and Aryl Ketones with H3PO2 or H3PO3: Its Application to the Synthesis of a Potential Anticancer Agent | Org. Lett. 2011; 13 (19): 5220
Impact of farnesylation inhibitors on survival in HutchinsonGilford progeria syndrome | Circulation 2014;130(1): 27
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B | N Engl J Med
. 2023; 388(8): 706
Etranacogene dezaparvovec for hemophilia B gene therapy | Therapeutic Advances in Rare Disease. 2021;2. doi: 10.1177/26330040211058896
Structureactivity relationship study to improve cytotoxicity and selectivity of lonafarnib against breast cancer cells | Arch Pharm (Weinheim) 2023; 356(4): e2200263
Deloitte | Measuring return from pharmaceutical innovation 2022
IQVIA | Global Trends in R&D 2021

Some recommended books on organic synthesis:
Clayden, Greeves, Warren; Organic Chemistry (basic organic chemistry knowledge)
Wyatt, Warren; Organic Synthesis: The Disconnection Approach (excellent introduction to retrosynthesis)
Kurti, Czako; Strategic Applications of Named Reactions in Organic Synthesis (extensive toolkit of functional group reactions and applications thereof with common conditions)
Nicolaou; Classics in Total Synthesis 13 (the ultimate total synthesis trilogy)
Nicolaou; Molecules That Changed the World (the world's most important molecules and their impact on everyday life)
Carreira, Kvaerno; Classics in Stereoselective Synthesis (compilation of the groundbreaking methods of stereoselective synthesis and application to synthesis of stereochemically complex structures)

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Pharmaceutical industry, worlds most expensive drug, chemical synthesis, ultrarare disease, Hemgenix, gene therapies, Skysona, Zynteglo, Institute for Clinical and Economic Review, costeffectiveness analyses, Soliris, research & development costs, pharma R&D, peak sales, market model, selling and marketing, grosstonet , US healthcare system, probability of success