From Bamboo Roots to Genetic Breakthroughs: Why Both CRISPR Therapeutics and NTLA Deserve a Spot in Your Portfolio
Unlike my all-time favorite in the AI space—Palantir—gene editing pioneers like CRISPR Therapeutics (CRSP) and Intellia Therapeutics (NTLA) have taken a quieter, more patient path. Their growth is less a sudden rocket and more akin to the bamboo: silent, persistent, and soon, ready to shoot skyward.
I started paying serious attention to this field after
reading The Code Breaker by Walter Isaacson, a gripping account of
Jennifer Doudna and the birth of CRISPR technology. It struck me how gene
editing is a lot like patching the code of an operating system—sometimes you
fix a glitch, sometimes you rebuild from the root. But here, the “software” is
human life itself.
So why write about it now? Because just like bamboo, the
moment when growth bursts into view is near. The recent, highly publicized
“failed” trial at Intellia wasn’t a setback, but a historic leap. It marked the
world’s first true gene insertion attempt inside a human body—a stepping stone
that every innovator knows paves the road to future breakthroughs.
Why CRISPR Therapeutics Stands Tall
CRISPR Therapeutics is a biotech leader wielding the
now-famous “genetic scissors”—the CRISPR/Cas9 platform—to fix or silence faulty
genes.
- Ex
Vivo Pioneers: Their flagship therapy (CTX001, branded as CASGEVY®) is
the world’s first FDA-approved CRISPR treatment, curing sickle cell
disease and β-thalassemia by editing a patient’s stem cells outside the
body, then reinfusing them.
- Expanding
Horizons: Beyond blood disorders, CRISPR is scaling into cancer
immunotherapy (off-the-shelf CAR-T) and cardiovascular disease, targeting
conditions like high cholesterol with in-body (in vivo) approaches.
- Financial
Muscle: With nearly $1.7B in cash and a deep partnership with Vertex
Pharmaceuticals, CRISPR can fuel R&D for years—essential for surviving
the notorious biotech “valley of death.”
- Long-Term
Vision: They don’t just treat symptoms; they aim for cures, directly
targeting the genetic root cause.
If CRISPR is the steady hand, Intellia Therapeutics
is the audacious innovator taking CRISPR into uncharted territory—inside
the living body.
- First
In Vivo Human Editing: Intellia’s NTLA-2001 is the first-ever to use
CRISPR as a “one-and-done” IV therapy. No need to remove cells; just a
single dose directly edits the culprit gene in the liver to treat rare
diseases like ATTR amyloidosis.
- Smart
Focus: Their pipeline is laser-focused on liver diseases (like
hereditary angioedema), where results are clear, biomarkers are
measurable, and the clinical impact is immediate.
- Breakthroughs
from ‘Failures’: NTLA-3001, though discontinued, was the world’s first
human test of gene insertion via CRISPR in vivo. Even the attempt yields
invaluable data for the next wave of treatments.
- Solid
Foundation: Over $1B in cash and a robust partnership with Regeneron
positions Intellia for sustained innovation and commercialization.
CRISPR vs. Intellia – Pipeline & Focus
CRSP |
NTLA |
|
Ex Vivo (Blood) |
✔️ |
|
Ex Vivo (CAR-T) |
✔️ |
|
In Vivo (Liver) |
✔️ |
✔️ |
In Vivo (Cardio) |
✔️ |
|
Liver Rare Disease |
✔️ |
|
Oncology |
✔️ |
Where They Differ—And Why It Matters : Ex Vivo vs. In Vivo Delivery
Feature |
CRISPR Therapeutics (CRSP) |
Intellia Therapeutics (NTLA) |
Delivery |
Ex Vivo: Cells edited outside the body |
In Vivo: Gene edited directly in the body |
FDA Approval |
FDA-approved (CASGEVY®) |
First in vivo trial, late-stage clinical |
Disease Focus |
Blood, cancer, cholesterol |
Liver diseases (ATTR, HAE) |
Manufacturing |
Cell-processing facilities |
Lipid nanoparticle platform |
Key Partnership |
Vertex Pharmaceuticals |
Regeneron |
Timeline of Major Gene Editing Milestones
- 2012:
CRISPR discovered as a gene editing tool
- 2020: CRSP’s CTX001 first ex vivo patient dosed
- 2023:
FDA approval for CASGEVY®
- 2021:
NTLA’s NTLA-2001, first in vivo human gene editing
- 2024:
NTLA-3001, first in vivo gene insertion trial attempted
Metric |
CRSP (Q1 2025) |
NTLA (Q1 2025) |
Cash & Equiv. |
$1.69 billion |
$1.01 billion |
R&D Spend (2024) |
$510 million |
$370 million |
Cash Runway |
Late 2026 |
Mid 2026 |
Market Cap (May) |
~$10 billion |
~$8 billion |
Why Investors Need Both
The “bamboo moment” in gene editing will not be a
winner-take-all event. CRISPR Therapeutics and Intellia Therapeutics
offer complementary bets:
- Different
Modalities: CRSP’s ex vivo and NTLA’s in vivo strategies both unlock
massive market opportunities across different disease areas.
- Mutual
Risk Mitigation: Investing in both diversifies your exposure; if one
technology stumbles, the other may still succeed.
- Shared Knowledge, Accelerated Progress: Even when one “fails” (like NTLA-3001), the learning accelerates the whole field—and both companies have the cash and partnerships to keep moving forward.
Final Thought:
Just as bamboo’s growth is invisible until it suddenly surges, gene editing’s
breakthroughs are approaching a visible tipping point. The investor who
appreciates the quiet, persistent groundwork—and sees the value in both
ex vivo and in vivo innovation—will be well positioned for the rewards when the
“bamboo shoots” finally burst above ground.
The Figured Figs Team 🌱
Disclaimer: “This article is for
informational purposes only and does not constitute financial, investment,
legal, or tax advice. Readers are encouraged to consult a licensed professional
before making any financial decisions."
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