John Crowley - Biotech's Chief Evangelist - [Art of Investing, EP.15]

21 insights saved from this episode by @science

@science· Biology
The initial Pompe treatment made two adult patients weaker, prompting Amicus to stop the trial and request an FDA clinical hold.
@science· Biology
A German infant failing approved Pompe therapy improved on Amicus’s experimental treatment, leaving the hospital breathing independently and feeding himself.
@science· Biology
The failed Pompe drug weakened patients because its half-life in human muscle differed from its half-life in animals and healthy volunteers.
@science· Biology
The Pompe enzyme treatment normalized the children’s enlarged hearts within six infusions or 12 weeks, preventing fatal cardiac decline.
@science· Biology
In the 1990s, science and biopharma transformed HIV/AIDS from a death sentence into a manageable long-term condition.
@science· Biology
A doctor predicted a Pompe disease cure within about a year. Nearly 25 years later, that goal remained unmet.
@science· Biology
About 90% of drugs entering clinical trials never become commercial products, while about 90% of successful products miss sales expectations.
@science· Biology
An angel round raised $1 million from 37 individual investors after the minimum investment fell from $250,000 to $5,000.
@science· Biology
Amicus’s global pivotal Pompe study enrolled about 120 people and cost more than half a billion dollars.
@science· Biology
One failed clinical study and the loss of a major pharmaceutical partner can threaten a biotech’s survival.
@science· Biology
Within six months of its UK launch, the Pompe medicine reached over 30 to 40 percent of people living with Pompe disease.
@science· Biology
Planning too far ahead can steer you toward a useful field you dislike, while life later invalidates the plan.
@science· Biology
In biotech, brilliant teams, advanced facilities, and massive funding still fail when the core technology fails.
@science· Biology
A résumé establishes baseline technical qualifications. The real hiring decision comes from how you spend the remaining time with the person.
@science· Biology
Going public brought capital but exposed the company to severe pressure managing a micro-cap biotech during the financial crisis.
@science· Biology
AI and machine learning could make drug screening and clinical trials faster, cheaper, less redundant, and more successful.
@science· Biology
A life-saving breakthrough can still be inadequate. The obligation is to return to the drawing board for something better.
@science· Biology
Education develops people through moral, mental, and physical principles, not merely through coursework.
@science· Biology
You do not need to be a scientist to invest in biotech. You need access to people who are.
@science· Biology
Long-term biotech investing starts with deep diligence on leadership, character, experience, and resilience.
@science· Biology
Better healthcare requires both newer treatments and a serious commitment to bringing them to everyone who needs them.

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