CRISPR Therapeutics (NASDAQ: CRSP) already holds one approved medicine and is racing to build a pipeline that could reshape the biotech landscape by 2036.
The company’s only approved therapy, Casgevy, treats sickle cell disease and transfusion-dependent beta thalassemia through a collaboration with Vertex Pharmaceuticals (NASDAQ: VRTX).
Vertex sells the medicine and takes 60% of the profit, leaving CRISPR with the remaining share while it continues to fund an ambitious research agenda.
Second-quarter 2026 revenue came to $10.2 million, set against a net loss of $91.2 million, with research spending driving much of that deficit.
Casgevy currently requires cell collection, lab-based editing, chemical treatment to clear the bone marrow, and reinfusion at a transplant center, making it a logistically demanding treatment to administer.
CRISPR’s strategy is to move gene editing inside the patient’s body entirely, converting that complex workflow into a standardized infusion product available at far more locations.
The company believes that shift would expand the pool of addressable patients worldwide from 150,000 to as many as 400,000 people.
A separate technical initiative called SyNTase editing aims to correct a mutation in a living organism rather than simply switching a dysfunctional gene off, which could open entirely new categories of treatable disease.
The pipeline program built around SyNTase, CTX460, targets alpha-1 antitrypsin deficiency and entered its phase 1 trial this year, with approval timelines typically averaging around 10.5 years for phase 1 assets.
Another program, CTX310, targeting severe hypertriglyceridemia, is currently in phase 1b, with an addressable patient population exceeding 1 million in the United States when counting refractory cholesterol disorders alongside the primary condition.
The path to 2036 could unfold across several distinct scenarios, ranging from modest disappointment to a genuinely landmark moment for medicine and for investors.
In a less favorable outcome, CRISPR could face the same challenge Novartis (NYSE: NVS) recently encountered, where a gene-editing candidate successfully altered biomarkers but failed to deliver the expected clinical benefit.
That scenario, applied across multiple disease contexts, could limit CRISPR’s growth trajectory even while the underlying technology continues to prove its editing capabilities.
A more likely base case sees two or three new therapy approvals by 2036, potentially pushing the stock to several multiples of its current value as revenue scales meaningfully beyond today’s levels.
The most optimistic outcome would involve precise stem cell editing techniques working inside living patients, a development that would push gene editing well beyond its current boundaries and make the upside for investors genuinely difficult to predict.
Even accounting for the risks and the long timelines involved, CRISPR Therapeutics remains a compelling long-term bet for investors comfortable holding through a decade of scientific progress and inevitable setbacks.
