We all now live in an era of great loss, but each loss is personal

Image for post
Image for post

The virologist who founded our firm closed our office ahead of the curve.

We packed up our laptops and notebooks. The pessimistic among us, sensing this would not be a short hiatus, took their plants. By a window overlooking Boston, my orchid withers.

Our endeavor is to create and finance new biotechnologies. My focus—having previously led a cancer therapeutics startup—is to help start new companies based on recent scientific discoveries.

Yet for a craft so linked to biology (molecules, organs, human life) our work is remarkably distant from the physical world. We are not battlefield heroes like nurses, physicians or grocery clerks. Aside for a colleague who moonlights as an ER doc, we assume no physical risk. …


Joining RA Capital in Boston to develop new biomedicines

Logo for RA Capital.
Logo for RA Capital.

I’m excited to say that I’ve joined the venture team at RA Capital, an approximately $2.5 billion biotech firm in Boston.

Do you have a compelling new project? Email me.

After closing up Nivien last fall, I returned to Harvard for my final semester. I took a neuro seminar, electromagnetism, drug liability litigation at Harvard Law, and a killer course in computational bio, graduating with a B.A. in Molecular & Cellular Biology.

The day after my last exam, I flew to Sydney to start a 6-month trip around the world. Along the way, I learned about and wrote essays on cybersecurity in biotech, drug pricing and rebates, delivering gene therapy, a novel cancer target, the path to power in pharma, and a guide on how to learn biotech. …


How hackers stole data from my biotech startup by infiltrating a global R&D contractor

I was on an island in Vietnam when I got the alert from the CEO of Charles River Labs, the world’s top R&D contractor: “highly sophisticated and well-resourced intruders” had stolen data from 1% of its clients — including my biotech startup, Nivien Therapeutics.

Image for post
Image for post

Charles River is a natural target: it handles data from startups like Nivien to giants like Pfizer, which enlist contractors for specialized expertise, capital-intensive infrastructure and experiments that exceed in-house bandwidth.

We worked with several contractors at Nivien: on animal studies, chemical screens, assay development and optimization of our therapeutic candidates. …


The scientists and startups hunting ROR1

Image for post
Image for post

Unlike common cancers of older age — liver, pancreatic, lung — blood cancers can get you early.

Leukemia and lymphoma emerge from your bone marrow or your lymphatic system and into your bloodstream, where the thumps of your heart drive the malignant cells throughout your body.

Fatigue, weakness, swollen nodes—by the time you notice, it’s often too late.

ROR1 is a promising new therapeutic target: a protein abundant on the surface of cancer cells but rare on healthy cells. A wave of experimental drugs exploit this difference to kill cancer without harming normal tissue.

Scientists at several startups and research institutes are pursuing four key strategies to bring ROR1-targeted therapies to patients. …


Midwest Mayor. Navy Lieutenant. Gay Episcopalian. Harvard Grad. Rhodes Scholar. Next President?

Image for post
Image for post

When Pete Buttigieg became Mayor of South Bend, Indiana, Newsweek had just featured his home as one of “America’s Top 10 Dying Cities.” South Bend embodied the same economic trends that went on to spawn Trump: rising unemployment, decaying social bonds, and anti-globalism.

Pete started by repairing roads and pledging to redevelop 1,000 vacant homes in 1,000 days. His team finished two months early. He lifted the minimum wage, recruited companies to open new offices, and invested in a major downtown revitalization while weathering multiple climate catastrophes.

Over eight years as Mayor, Pete transformed the struggling manufacturing community into a Midwestern tech hub. Today, South Bend is a prosperous city with a growing youth population, 3.6 …


How scientists used genetic engineering and ML to bring a virus back from the dead as a better deliver method for gene therapies

Image for post
Image for post
Molecular structure of the Anc80 virus. [Figure 2C, Zinn et al. 2015]

Gene therapy is a powerful tool to treat diseases from cancer to deafness, but it requires safe and effective delivery to the correct cells in the body. This post is about the unique genesis of Anc80, a new viral delivery system.

Gene therapy has two parts: 1) the genetic package, to fix a disease-causing error, and 2) the delivery system, to deliver the fix to its intended location.

Existing delivery systems cannot reach all cells, limiting gene therapy’s utility.

Anc80 has proven 1000X more effective in targeting difficult-to-reach cells in mice and the first human trials start this year. …


Killing PBM rebates could lower drug prices without hurting R&D

Update: The Trump Administration withdrew this HHS proposal on July 10th, 2019.

Image for post
Image for post

People are paying more for drugs, even as inflation and production costs fall. However, many politically-popular tactics to lower prices could hit R&D budgets — the lifeblood of new medicines.

In a proposal to lower prices out tonight, the Dept. of Health and Human Services (HHS) instead goes after “significant distortions in the drug distribution chain” that increase out-of-pocket costs.

The HHS solution: kill the sketchy rebates drug companies pay to pharmacy benefit managers (PBMs) for higher placement on formularies.

Already confused? That’s what PBMs want. …


The best in scientific literature, books, essays, news, newsletters, government resources, podcasts, forums, analysts, and more!

Image for post
Image for post

This is a roundup of top resources in biotech, pharma and healthcare.

My list excludes many great ways to learn about biotech: conferences, scientific degrees (and textbooks), talking with people (from professors to executives), or actually working in the space.

However, it’s the most detailed and now most widely-read set of materials you can access at any time, from anywhere in the world, mostly for free.

If I’ve missed a good source or you’d like to connect, feel free to email me.

Scientific Literature

PubMed is the bread-and-butter of biotech knowledge. If you feel daunted by the volume of content, try this real-time list of trending papers. …


Image for post
Image for post
The FDA is approving more drugs for rare diseases each year than ever before.

When politics, climate change or international conflicts have me down, I turn to biotech instead. There’s always a new scientific discovery, a new drug for a rare disease, a new medical device for a common ailment.

But non-industry media under-reports good news in biotech. Many biomedical stories that do go mainstream are inaccurate.

Here are ten real biomedical wins from 2018 (with a contentious #10):

  1. Alnylam* won approval for the first drug that uses RNAi, a new modality to silence mutations that cause genetic diseases like hATTR amyloidosis.
  2. Catalent won approval for a novel opioid-withdrawal treatment, a critical success near to my heart in the midst of a relentless American opioid crisis. …


A Study of CEOs at the Top 50 Biopharmaceutical Companies

Skip to the end for key highlights and analysis.

Image for post
Image for post

Biopharma leaders allocate billions in R&D funding. They decide which diseases will be studied, treated, and one day cured — and which will not.

They control drug prices, and therefore access to treatments for everything from cancer to diabetes. And they earn among the highest salaries of all executives, yet often go unnoticed compared to leaders in tech or finance.

Over the course of a life everyone uses biopharma products, but there are no good summaries of the CEOs at each of the top 50 biopharma companies.

What path did each CEO follow — or blaze — to reach such a vital role in the discovery, development and distribution of life-saving technologies?

About

Nathaniel Brooks Horwitz

Biotech VC. Director or Observer on the boards of six RA Capital companies. Former CEO of Nivien Therapeutics. My writing does not represent RA Capital.

Get the Medium app

A button that says 'Download on the App Store', and if clicked it will lead you to the iOS App store
A button that says 'Get it on, Google Play', and if clicked it will lead you to the Google Play store