O CRISPR Decoded

Treatments

Named gene-editing and gene-therapy medicines, from approved products to programmes that have been discontinued.

15 treatments in CRISPR Decoded. Grouped by the stage each programme has actually reached. Only approved products are available to patients outside a trial; “in trials” never means a treatment is proven.

15 exibidos

Approved3

Casgevy (exagamglogene autotemcel) Vertex Pharmaceuticals / CRISPR Therapeutics Approved 2023 · $2.2 million (US list price) CRISPR-Cas9 Lyfgenia (lovotibeglogene autotemcel) bluebird bio Approved 2023 · $3.1 million (US list price) Lentiviral gene addition Zynteglo (betibeglogene autotemcel) bluebird bio Approved 2022 · $2.8 million (US list price) Lentiviral gene addition

Phase III4

Nexiguran ziclumeran (nex-z) Intellia Therapeutics CRISPR-Cas9 Lonvoguran ziclumeran (lonvo-z) Intellia Therapeutics CRISPR-Cas9 Risto-cel (BEAM-101) Beam Therapeutics Base editing BEAM-302 Beam Therapeutics Base editing

Phase II1

VERVE-102 Eli Lilly Base editing

Phase I4

PM359 Prime Medicine Prime editing CTX112 CRISPR Therapeutics CRISPR-Cas9 CB-010 Caribou Biosciences CRISPR-Cas12a Hypoimmune islet cell therapy Sana Biotechnology CRISPR-Cas9

Preclinical1

EDIT-401 Editas Medicine CRISPR-Cas9

Not proceeding1

EDIT-101 (Brilliance) Editas Medicine CRISPR-Cas9

Other status1

Bespoke base editor for CPS1 deficiency (the KJ Muldoon case) Academic: CHOP and Penn Completed

Como ler este painel Apenas a primeira coluna foi autorizada por um regulador para uso em pacientes. Tudo à direita dela é pesquisa: estar na Fase III não é evidência de que um tratamento funciona, e a maioria dos programas em cada etapa nunca chega à aprovação. As etapas refletem o estudo mais avançado para o qual temos um registro.