Gene-editing stocks took a big step forward in November when CRISPR Therapeutics (CRSP 3.79%) reported fairly positive results from the first patients treated with a CRISPR-based drug. Early success from the present leader in the field also pushed up shares of Editas Medicine (EDIT 3.88%) and Intellia Therapeutics (NTLA 6.38%), even though neither has reported any clinical-trial results yet.
If you missed out on the latest big catalyst that drove all three CRISPR stocks higher, don't worry -- you'll get more chances soon. Here's what you need to know about the road ahead for CRISPR, Editas, and Intellia.
Image source: Getty Images.
Intellia Therapeutics: Bringing up the rear
The first new drug candidates from CRISPR and Editas really aren't very far apart on the development timeline, but Intellia is miles behind both its peers. This straggler hasn't even submitted its first investigational new drug (IND) application, which the Food and Drug Administration (FDA) must approve before the company can begin testing a drug in humans.
Intellia doesn't think it will be able to submit its first IND until the middle of 2020. If the FDA greenlights clinical-stage testing with the company's lead candidate NTLA-2001 for the treatment of transthyretin amyloidosis, Intellia could begin treating patients before the end of next year. It's way too early to pin down a date, but we won't be able to look at clinical data until the first half of 2021 at the earliest.
Editas Medicine: Getting closer
This stock's next big catalyst isn't as far away as Intellia's, but isn't exactly around the next corner. Editas Medicine sent in an IND application in 2018 for its lead candidate, a blindness prevention drug called EDIT-101, but didn't begin screening patients until the middle of 2019.
Editas doesn't intend to dose anyone with EDIT-101 until early 2020. Investigators are going to observe patients for an entire year following a single injection of the gene therapy, which means the first complete results won't be ready until mid-2021 at the earliest.
While the study calls for observation one year after an infusion with EDIT-101, interim assessments could give Editas stock a big boost before the end of 2020.
Image source: Getty Images.
CRISPR Therapeutics: More than a name
Name recognition was largely responsible for driving this stock higher than its peers around 1 1/2 years ago. CRISPR Therapeutics still sports a market value larger than Editas and Intellia combined, but now there's a good reason.
CRISPR Therapeutics' lead candidate CTX001 is the only CRISPR-based candidate that's been used to treat patients, and recently released results were encouraging. With help from deep-pocketed partner Vertex Pharmaceuticals, CRISPR's running two studies with its lead candidate Climb THAL-111 for patients with transfusion-dependent beta-thalassemia and Climb SCD-121 for people with severe sickle-cell disease.
Four months after receiving a single infusion of CTX001, the patient in Climb-SCD hadn't experienced any blood-vessel blockages. It's been nine months since the Climb-THAL study began, and this patient hasn't required any blood transfusions.
CRISPR hasn't set a date for the next observation, but both trials are designed to follow patients for two years. That means there will be plenty of potential catalysts from the CTX001 program scattered throughout the next several years.
CRISPR Therapeutics has also started treating patients with CTX110, which is essentially a bunch of immune cells trained to kill cancer cells with CD19 on their surface. Unlike existing treatments that take weeks to modify a patient's own blood cells, CTX110's a one-size-fits-all solution so oncologists can begin treatment immediately.
We'll probably get to look at results from CRISPR's oncology program in the first half of 2020. Around the same time, the company expects to begin dosing patients with a similar treatment, CTX120, which targets BCMA-presenting cancer cells.
Image source: Getty Images.
No guarantees
It's still too early to predict success for any of these upcoming CRISPR-stock catalysts. They could jump much higher, but there aren't any guarantees. We've seen interim results from just two patients treated with a CRISPR-based drug, and those patients aren't even halfway through their respective studies.
These companies are going in different directions, but they're all depending on six-figure price tags for single-administration therapies. If the impressive efficacy results we've seen from the first patients treated so far aren't durable through two years or longer, all three of these stocks could tank.
