Develops gene-edited medicines and cell therapies for people with blood disorders, cardiovascular disease, cancer, autoimmune diseases and diabetes.
10-K for FY2025, filed Feb 12, 2026Our mission is to create transformative gene-based medicines for serious human diseases. We are a leading biopharmaceutical company focused…View ↗
We have established a portfolio of therapeutic programs spanning four core franchises: hemoglobinopathies, in vivo, CAR T approaches and…View ↗
Hemoglobinopathies: Our most advanced program, CASGEVY, has received approval in the United States and other countries for the treatment of…View ↗
In vivo approaches: We are advancing a portfolio of programs leveraging in vivo editing for both common and rare diseases, as well as using…View ↗
CAR T: We are progressing next-generation gene-edited cell therapy programs, including allogeneic chimeric antigen receptor T cell, or CAR…View ↗
Regenerative medicine: We are advancing a deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet…View ↗
The revenue policy describes payments for contracted goods and services, with timing depending on the work: customers may pay as services are provided or when a product or service is delivered. Collaboration arrangements are outside that policy.
10-K for FY2025, filed Feb 12, 2026A contract with a customer exists when (i) the Company enters into an enforceable contract with a customer that defines each party’s rights…View ↗
5) Recognize revenue when or as the Company satisfies a performance obligationView ↗
The Company satisfies performance obligations over time or at a point in time, depending on the nature of the performance obligation.…View ↗
10-K for FY2025, filed Feb 12, 2026
FY2025 revenue by product or service ↗$3.5M
10-K for FY2025, filed Feb 12, 2026
$3.5M ↗| Segment | Share | Revenue |
|---|---|---|
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Collaboration Revenue
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0.0%
FY2022: 36%
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Collaboration Revenue, FY2025
$0 Sources
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|
Grant Revenue
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100%
FY2022: 64%
|
Grant Revenue, FY2025
$3.5M Sources
|
The company works with Vertex on the development and commercialization of CASGEVY, and with Sirius on siRNA research, development and commercialization. It also collaborates with Eli Lilly on zugo-cel clinical evaluation.
10-K for FY2025, filed Feb 12, 2026CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy, in which a patient’s own hematopoietic stem and progenitor cells are…View ↗
Zugo-cel continues to advance in both autoimmune disease and hematologic malignancies. In autoimmune disease, it is being investigated in…View ↗
siRNA. In May 2025, we partnered with Sirius and entered into the Sirius Agreement pursuant to which, among other things, we and Sirius…View ↗
No. It lost $581.6M in FY2025.
10-K for FY2025, filed Feb 12, 2026
−$664.6MView ↗
10-K for FY2025, filed Feb 12, 2026
−$581.6MView ↗
10-K for FY2025, filed Feb 12, 2026
−$345.9MView ↗
10-Q for Q2 FY2026, filed Aug 3, 2026
−$91.2MView ↗
10-Q for Q2 FY2026, filed Aug 3, 2026
Open tableView ↗
8-K, filed Aug 3, 2026
CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT that has been shown to reduce…View ↗
Shrinking. Revenue fell 100% in 4 years, from $915M in FY2021 to $3.5M in FY2025, about 75% a year.
New product and research areas
The older report described a portfolio centered on hemoglobin disorders, cancer cell therapies, diabetes cell replacement and in vivo editing. The latest report also describes siRNA medicines and autoimmune cell therapy programs.
10-K for FY2022, filed Feb 21, 2023 10-K for FY2025, filed Feb 12, 2026siRNA-based ProgramsView ↗
Our siRNA-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with…View ↗
We believe CRISPR/Cas9 has the potential to create the next generation of CAR T cell therapies that may have a superior product profile and…View ↗
Zugo-cel continues to advance in both autoimmune disease and hematologic malignancies. In autoimmune disease, it is being investigated in…View ↗
Competes with Editas Medicine, Intellia Therapeutics, Metagenomi, Scribe Therapeutics, Beam Therapeutics, Prime Medicine, Tessera Therapeutics, Verve Therapeutics, Allogene Therapeutics, Cellectis, Iovance Biotherapeutics, Factor Bioscience, Korro Bio, nChroma Bio, Precision BioSciences, Sangamo Therapeutics, Tune Therapeutics, Wave Life Sciences, 4D Molecular Therapeutics, and Sarepta Therapeutics
10-K for FY2025, filed Feb 12, 2026Our platform and product focus is on the development of therapies using CRISPR/Cas gene editing technology. We are aware of several…View ↗
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