Therapeutic area
Rare Disease biotech companies
The 16 rare disease 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 |
|---|---|---|---|---|
| VRTXVertex Pharmaceuticals | Trikafta · cystic fibrosisApproved | $13.64B2026-06-30 | — | maintained |
| MRNAModerna | mRNA-1345 (RSV) · Respiratory syncytial virus (RSV) in older adultsApproved | $6.91B2026-06-30 | — | archived |
| ALNYAlnylam Pharmaceuticals | Amvuttra · ATTR amyloidosisApproved | $3.31B2026-06-30 | — | archived |
| IONSIonis Pharmaceuticals | Tryngolza · familial chylomicronemia syndromeApproved | $2.70B2026-06-30 | ~71 mo | archived |
| CRSPCRISPR Therapeutics | Casgevy (exagamglogene autotemcel) · Sickle cell disease and transfusion-dependent beta-thalassemiaApproved | $2.36B2026-06-30 | ~74 mo | maintained |
| NTLAIntellia Therapeutics | NTLA-2001 (nexiguran ziclumeran) · Transthyretin amyloidosis (ATTR) — hereditary and wild-typePhase 3 | $628M2026-06-30 | ~19 mo | maintained |
| RAREUltragenyx Pharmaceutical | Crysvita · X-linked hypophosphatemiaApproved | $436M2026-06-30 | ~9 mo | archived |
| ACADACADIA Pharmaceuticals | Nuplazid · Parkinson disease psychosisApproved | no filing parsed | — | archived |
| BEAMBeam Therapeutics | BEAM-101 · sickle cell diseasePhase 1 | no filing parsed | — | archived |
| BIIBBiogen | Leqembi · Alzheimer diseaseApproved | no filing parsed | — | archived |
| BMRNBioMarin Pharmaceutical | Voxzogo · AchondroplasiaApproved | no filing parsed | — | archived |
| DYNDyne Therapeutics | DYNE-101 · myotonic dystrophy type 1Phase 2 | no filing parsed | — | archived |
| PVLAPalvella Therapeutics | PTX-022 (QTORIN sirolimus) · Microcystic Lymphatic MalformationsPhase 3 | no filing parsed | — | archived |
| PRTAProthena | Birtamimab · AL amyloidosisPhase 3 · stopped | no filing parsed | — | archived |
| RXRXRecursion Pharmaceuticals | REC-994 · cerebral cavernous malformationPhase 2 | 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
- 2026-12 · IONS · Donidalorsen OLE: Donidalorsen in Hereditary angioedema prophylaxis — primary completion ClinicalTrials.gov
- 2027-01-25 · VRTX · Suzetrigine DPN open-label extension: Suzetrigine in Diabetic peripheral neuropathic pain — primary completion ClinicalTrials.gov
- 2027-04-06 · VRTX · ELX/TEZ/IVA non-F508del: Elexacaftor/tezacaftor/ivacaftor in Cystic fibrosis, non-F508del genotypes — primary completion ClinicalTrials.gov
- 2027-05-31 · SRPT · ENVISION: Delandistrogene moxeparvovec in Duchenne muscular dystrophy — primary completion ClinicalTrials.gov
- 2027-05-31 · VRTX · Suzetrigine DPN Phase 3: Suzetrigine in Pain associated with diabetic peripheral neuropathy — primary completion ClinicalTrials.gov
- 2027-08 · ACAD · ACP-211 MDD: ACP-211 in Major depressive disorder with inadequate response — 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
- 2027-12-27 · ALNY · TRITON-PN: Nucresiran in Hereditary ATTR amyloidosis with polyneuropathy — primary completion ClinicalTrials.gov
- 2027-12-30 · BEAM · BEAM-301 GSDIa: BEAM-301 in Glycogen storage disease type Ia — primary completion ClinicalTrials.gov
- 2028-01 · ACAD · ADVANCE-2 master protocol: ACP-204 in Alzheimer's disease psychosis — primary completion ClinicalTrials.gov
- 2028-01 · RXRX · ELUCIDATE: GTAEXS617 in Advanced solid tumours — 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.