🌊 Gene Editing

Gene Editing: why now, what the market looks like and how to surf it.

Oct 7, 2022

Hello! I’m Ivan Landabaso, partner at JME.vc. Join >4.5K entrepreneurs surfing startup trends. Once or twice per month. From Spain to the 🌍

Summary

Gm startup riders! This week’s good stuff for your startup brain includes:

🧬 Gene Editing: why now and what the market looks like.
🍬 Startup Candy: Glovo & Cabify multiplier effect, robotics and prioritisation.
💵 Deals & Jobs: 25 startup deals in Spain (>€110M).

🧬 Gene Editing

This week my good friend Philippe Koch - cellular and molecular biologist currently at ZS - takes us down the Gene Editing rabbit hole. Lets go:

😟 Problem

For as long as we’ve been getting sick, we’ve been trying to cure the different conditions and diseases that impact us. Unfortunately, many treatments fall short of this goal and instead focus on managing the symptoms rather than the underlying cause.

🤩 Solution

Cell & gene therapies aim to do just that; science has found ways to correct the underlying errors in our genetic building blocks or even program our body’s ability to fight diseases … There are treatments out there that provide “cures” (any big statement like that comes with disclaimers, more on that below…)

⏳ How we got here

The concept of using cell and gene therapies isn’t new – in fact, we’ve already seen a cell and gene therapy revolution of sorts in the 90s when, for the first time ever, we successfully alleviated a person’s genetic defect (think error in your body’s operating system) by introducing a correct and functioning version of that gene into their body.

Whilst the effects were not permanent, this was an exciting milestone that saw new technologies quickly developed.

Inspired by viruses (hate to say it but think covid) we managed to harness their unique ability of hacking our operating systems to make us produce more copies of themselves. In doing so, we created “good” viruses that could hack and insert the right genes into our operating system.

Unfortunately, this success was short lived as a patient died due to complications which put a pause on things as we realized we needed to go back to the drawing board…

Over the years research went into refining other viral delivery methods and therapies such as CAR-Ts; this last one being especially cool as this cell therapy essentially programs T-Lymphocytes - think the Robocops of your blood - to become better at detecting and destroying cancer. Whilst this was exciting, progress was relatively slow until…

CRISPR came along:

The Ultimate Guide To CRISPR: Mechanism, Applications, Methods & More — Source

CRISPR was first discovered as scientists studied how bacteria protected themselves against viruses, from there it was then adapted to be applicable in humans picking up the 2020 Nobel prize in chemistry along the way.

CRISPR essentially gives researchers the ability to FIND and REPLACE faulty genetic code with the correct sequence… that’s like being able to pick out that spelling mistake on page 96 of your body’s operating manual.

Pretty neat, no? Not so fast! This technology is still somewhat in its infancy and a lot of work is going into making sure that no ‘off-target’ replacements are made… think about it - how many times have you used FIND/REPLACE in a word or ppt presentation as a lazy way to bring in some last-minute revision only to find whole sentences now jumbled throughout the document? Now think about the consequences if you did that to your body’s operating manual…

We are at a point where CRISPR technology can be used not only to treat conditions but even to engineer super humans. A couple of years back, a scientist announced he had genetically engineered human embryos to be HIV resistant - whilst on the face of it that might seem like a worthwhile cause it does open a host of ethical dilemmas which I won’t get to here … suffice it to say the (now disgraced) scientist in question also had plans of launching a “genetic medical tourism” company.

🗺️ Market Map

For those that want to get geeky with it, Cell and Gene therapies can be bucketed into different categories. True to their name, you can have cell-based therapies (such as the CAR-Ts treatments mentioned above) that either rely on harvesting your own cells (Auto=self) or come from a donor (Allo=other).

To complicate matters, you can enhance these cell-based treatments with Gene editing tools such as CRISPR (ex-vivo = outside your body) OR you can also go the direct route and receive the Gene Therapy straight into your body (in-vivo = inside your body).

Check out the below visual as a recap (courtesy of my employer: ZS Associates)

The field of cell and gene therapy is expected to absolutely boom with a lot of companies that are in clinical trials right now - by 2025 we’d expect to have around 30 gene and an additional 30 cell therapies on the market. Compared to the handful of therapies on the market right now, that’s an insane amount of expected growth!

Check out the below graphic for some more details:

Amongst others, some of the main challenges cell and gene therapies are facing are:

1. Patient access - making sure that everyone has equal access to these treatments is tricky due to a number of reasons which include - specific genetic testing is not widespread, these advanced therapies can generally only be obtained at big academic hospitals and the reimbursement is complex (and still being defined)

2. Manufacturing - cell and gene therapies are complex and laborious to make so this is becoming a real bottleneck that manufacturers are having to solve. Fun fact I heard at a recent conference: apparently baristas and bakers make up most of the talent pool that’s filling the current staffing shortage at manufacturing facilities - must be something about being able to follow detailed recipes!

🚀 My favorites

Novartis Gene Therapies - Zolgensma. Transforms the course of a disease in children that would have almost certainly died by the age of 2.
GenSight – Whilst still in clinical trials – GenSight is looking into using a gene therapy to restore vision.
Matricelf – Also still very much clinical, but combines 3D printing with cell therapies to repair a person’s heart after they have had a heart attack
A special shoutout also to GenMab, who are located right around the corner in Utrecht, who are doing some amazing work to develop specialized antibodies for the treatment of cancer

👀 Watch out

Notice how I said “Cure” earlier? How often have you seen that super click-baity article that claimed that “We’ve cured cancer!”?.... and yet most of us will still be or have loved ones be impacted by cancer.

So where’s this cure then? As always, the answer is, it’s complicated:

The term cancer describes of host of conditions that manifest in the uncontrollable replication of cells - problem is there’s a huge number of different types of cells in our bodies and an even bigger number of things that can cause this so there really isn’t a “one size fits all” type of solution (at least for now)
When we say “cure” that’s to the best of our knowledge - we simply don’t have the long-term data that would indicate otherwise. Whilst the hope is that a cell or gene therapy’s durability will last a lifetime, this technology is simply too young for us to say that with 100% certainty. Not only is that a stark reality check for any patients that have been “cured” but it also opens up some interesting reimbursement questions especially when the one of the reasons for the eye watering prices of these treatments (think up to multimillion $ / treatment) is their “curative” properties

🐇 Follow the White Rabbit 🕳️

ZS Insights: I work with ZS Associates where I’ve been lucky enough to work with a bunch different Cell and Gene therapy companies at different commercial stages - that gives us a unique perspective in this space.
The BBC made an amazing documentary that follows the lives of 2 patients undergoing cell therapies - it’s a sobering reminder that the field of cell and gene therapy, for all its advances, still has a ways to go and that there are a bunch of things we can improve beyond just the science to help patients (link)

🍬 Sartup Candy

💵 Startup Deals & Jobs

Deals

You love startups and want to enjoy a Spanish lifestyle? Come join the Spanish startup ecosystem. Here’s a list of recently funded startups:

Exoticca (traveltech) raised 20M
Cledara (saas) raised 20M
Toquio (fintech) raised 20M
Good News (coffee) raised 11M
Penpot (design) raised 8M
Metricool (analytics) raised 5M
PropHero (proptech) raised 5M
PandaGo (mobility) raised 4.7M - JME Ventures portfolio
Tuio (insuretech) raised 3M
Reveni (fintech) raised 3M - JME Ventures portfolio
PSquared (proptech) raised 1.6M
Innomylabs (footech) raised 1.3M
Cuyna (dark kitchens) raised 1.2M
Lisa (insuretech) raised 1.2M
Union Avatars (gaming) raised 1.2M
Tennders (logistics) raised 1.2M
Box2Box (logistics) raised 1.1M
Indya (sports) raised 1M
Vitruve (sports) riased 1M
Hotelverse (tourism) raised 1M
Lifecole (edtech) raised 750K
Ripfoods (foodtech) raised 600K
Nivimu (hr saas) raised 500K
Mixo (foodtech) raised 500K
Solfy (energy) raised 400K
Clickout raised 100K

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