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Molecular Minute: January 2022

Welcome to Molecular Ideas, and thank you for sharing your time with us! Our Molecular Minute series covers some of the other notable events in the life science innovation that we didn't get to discuss in the moment this month. While some topics may be covered in greater depth in future posts, we hope to provide an exciting overview of what's next.


January was an busy month for the life sciences. Boundaries were broken, justice was served, and hopes for the future of generic pharmaceutical access are becoming ever brighter. Plus, Moderna's mRNA vaccine was fully approved by the FDA, so welcome to the world of mRNA therapies! In our Molecular Minute series, we aim to briefly recap and share our takes on some of these notable events (with links for you to learn more).

Image Source: Media from Wix


When Pigs Fly

The need for viable organs to help people recover from disease or save lives is possibly one of the most significant and intractable healthcare burdens we face today. The technical, ethical, and clinical burdens of organ transplantation make it exceptionally difficult to navigate patient needs with the current supply of human organs on hand at any given moment.


While facing impossible odds, people with siblings or MBAs will tell you this: when you can't win the game you're playing, try to change the rules.


January 2022 marked the first successful transplant of a humanized pig heart into a human. The patient was unable to receive or unresponsive to other therapies. This pig heart had been 'humanized' through ten genetic alterations. Several existing pig genes were silenced and new genes were inserted to prevent the patient's immune system from rejecting the tissue. While it will take months - maybe years - to know how long this new heart will last, this represents an unforgettable landmark. Now it's up to Kiniksa Pharmaceuticals and their competitors to fulfill the hope this innovation brings to patients and families around the world.


As if the story wasn't wild enough - in order to help the heart beat successfully, the patient needed to be infused with a miniscule dose of cocaine (and hormones). This is not a joke. Though cocaine is still very illegal in the U.S., it was given special dispensation as part of a life-saving procedure. What are the odds?


Read more with: New York Times | NBC News | Nature | IFLScience


When Your Best Defense Leads to Defenestration

There are two ways to leave a relationship. The first is to do so quietly, respectfully, and politely. This shows a degree of mutual respect in a solemn moment after a trying time.


The second is to get chucked out a window. It's not always undeserved.


After years of litigation and possibly one of the worst public relations firestorms in pharmaceuticals, former CEO Martin Shkreli is banned from the industry for life. He also has to pay $64.6 million for violating antitrust laws (under the realm of "wrongfully obtained profits"). The outcry over the began in 2015, when the "Pharma Bro" raised the price for the anti-parasitic drug Daraprim® (pyrimethamine) from $13.50 to $750 overnight. Adding insult to injury, the drug is on the World Health Organization's List of Essential Medicines given its utility in treating major parasites like toxoplasmosis and malaria, as well as other diseases faced by immunocompromised patients. The defunct CEO is scheduled to be released from custody in 2023. Where he goes from there will be a mystery, but it won't be back to pharma.


Patients should not have to stand alone against their disease. While the maelstrom of exacting clinical standards, long investment horizons, and high capital requirements demand that new drugs yield profitable returns, the pharmaceutical industry is no place for profiteering. Profits mean your drug works, patients adhere to it, and (ideally), there are cost sharing mechanisms to ensure everyone can pay (and is paid) for it.


Ironically, in the same year, the CEO of Novo Nordisk said "If we wind up curing diabetes, and it destroys a big part of our business, we can be proud, and you can get a job anywhere." You can guess which company is still going strong.


Read more with: New York Times | Wall Street Journal | Washington Post


When There's Hope for Drug Access

Still, there is some good news in the world of improving patient access through lower drug pricing. The billionaire and legendary entrepreneur Mark Cuban launched an online pharmacy under the umbrella of helping patients gain access to critical generic drugs at lower prices. Price markups of drugs - even generics - cascade with each successive stakeholder interaction. As you may have guessed, there are a lot; distributors and wholesalers take notable cuts, and pharmacy benefit managers (PBMs) have created a black box for formulary (coverage) negotiations.


Enter Cost Plus Drug Company. As of this writing, price reductions range from hundreds to thousands of dollars. You can find a list with the most notable price cuts here. In true form, Mark Cuban made his mechanism for these savings easy to understand. Patients will be charged the manufacturer’s price for a drug, plus a flat 15 percent markup and pharmacist fee.


No complex derivations via backroom negotiations. It's worth pointing out that this isn't entirely new ground. Ro has had years of experience in delivering low-cost, branded versions of generics for specific health challenges (ranging from hair loss to smoking cessation to erectile dysfunction) through an easy-to-use API. Capsule has positioned itself as an Amazon for drugs, providing prescriptions with one-day delivery. Even major pharmacy chains like Duane Reade and CVS have portals to manage prescriptions.


Everyone claims to be cheaper, faster, and easier. Still, nothing beats the combined value of dollars saved and drug diversity, especially for chronic conditions. Mark Cuban and his team may be successful in blowing down the fun house of smoke and mirrors around generic pharmaceutical pricing - only time will tell.


Read more with: Wall Street Journal | Forbes | NPR


Bonus: Startup Spotlight

In this section, we shine a light on a pre-commercial biotechnology or medtech company with interesting and innovative technology. This month, we are pleased to highlight Gameto, a biotechnology company focused on female reproductive longevity.

What if women could make their reproductive biological clocks tick a little slower? That's the question at hand. The ovaries are essential to women's health - reproductive and otherwise as hormone producers. However, the ovaries age approximately five times faster than other organs in the body. It's an early-stage company, having just raised $20 million in Series A funding.


By reprogramming ovary cells using cell therapies, the company aims to begin by addressing menopause and improving reporductive fertility in three programs.


The social (and by extension, economic) impacts of a potential success cannot be overstated. With lifespans increasing and more women in the workforce (yay!), expanding the the window of fertility offers an unprecedented choice in life planning. That has the potential to reshape personal lives and entire economies alike.


That’s all for today! Thanks for spending your time with us. Please share this article, and sign up to leave your thoughts, ideas, and opinions in the comments. Your feedback is always welcome and helps Molecular Ideas grow!

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