Gene editing (rare disease)
In vivo editing platforms racing from liver-targeted rare diseases outward.
Lenses
EarlierApproval →
NTLAPh3cash ok
2026-10-30 (78d)Company-guided
In vivo CRISPR ATTR · NCT06128629 · Pivotal ATTR update
CRSPPh1cash ok
2027-01-25 (165d)Estimated
CTX310 in vivo · NCT06451705 · In vivo editing update
BEAMPh1cash ok
2027-03-20 (219d)Estimated
BEAM-302 AATD · NCT06389877 · AATD update
VERVPh1cash ok
2026-11-12 (91d)Company-guided
VERVE-102 · NCT06164730 · Dose / safety update