Therapeutic area
Cell & Gene Therapy biotech companies
The 4 cell & gene therapy companies we cover, with cash and runway from their latest SEC filings, trial milestones from ClinicalTrials.gov, and FDA decisions from their own 8-Ks. Companies are tagged by the areas their pipelines target.
Companies
| Company | Most advanced program | Cash & investments | Runway | Profile |
|---|---|---|---|---|
| CRSPCRISPR Therapeutics | Casgevy (exagamglogene autotemcel) · Sickle cell disease and transfusion-dependent beta-thalassemiaApproved | $2.36B2026-06-30 | ~74 mo | maintained |
| RAREUltragenyx Pharmaceutical | Crysvita · X-linked hypophosphatemiaApproved | $436M2026-06-30 | ~9 mo | archived |
| BEAMBeam Therapeutics | BEAM-101 · sickle cell diseasePhase 1 | no filing parsed | — | archived |
| SRPTSarepta Therapeutics | Elevidys · Duchenne muscular dystrophyApproved | no filing parsed | — | archived |
Runway is cash divided by quarterly operating burn and is not shown for companies funded by their own revenue. Figures and their dates are on each company page, linked to the filing.
Upcoming trial milestones
- 2026-10-31 · SRPT · MIS51ON: Eteplirsen (high dose) in Duchenne muscular dystrophy, exon 51 — primary completion ClinicalTrials.gov
- 2026-12 · BEAM · BEAM-201 T-ALL: BEAM-201 in Relapsed/refractory T-ALL or T-LL — primary completion ClinicalTrials.gov
- 2027-05-31 · SRPT · ENVISION: Delandistrogene moxeparvovec in Duchenne muscular dystrophy — primary completion ClinicalTrials.gov
- 2027-08 · RARE · MCE study: Triheptanoin in Long-chain fatty acid oxidation disorders — primary completion ClinicalTrials.gov
- 2027-09 · RARE · GTX-301: DTX301 in Late-onset OTC deficiency — primary completion ClinicalTrials.gov
- 2027-12-30 · BEAM · BEAM-301 GSDIa: BEAM-301 in Glycogen storage disease type Ia — primary completion ClinicalTrials.gov
- 2028-05 · BEAM · BEAM-302 AATD: BEAM-302 in Alpha-1 antitrypsin deficiency — primary completion ClinicalTrials.gov
A registry milestone is the date data collection is scheduled to end, not the date results are released.
FDA decisions
None of these companies has an FDA decision on our PDUFA calendar, which lists only dates a company has stated in a filing.
Educational information, not investment advice. See where each field comes from.