Rare genetic companies
41 listed rare genetic companies, largest first, with lead program and the next catalyst each has disclosed.
| Company | Ticker | Lead program | Indication | Next catalyst |
|---|---|---|---|---|
| Alnylam Pharmaceuticals, Inc. | ALNY | AMVUTTRA (vutrisiran) | hATTR amyloidosis with polyneuropathy; ATTR-CM | November 2026 — FDA target action date for NDA in generalized myasthenia gravis |
| Ascendis Pharma A/S | ASND | TransCon CNP (navepegritide) | achondroplasia in children | Q4 2026 — European Commission decision on marketing authorisation application |
| Biomarin Pharmaceutical Inc | BMRN | VOXZOGO (vosoritide) | achondroplasia; hypochondroplasia (sNDA) | February 28, 2027 — PDUFA target action date for sNDA (full approval, achondroplasia) |
| Krystal Biotech, Inc. | KRYS | VYJUVEK (beremagene geperpavec-svdt, B-VEC) | Dystrophic epidermolysis bullosa | 2026 — IOLITE Phase 3 top-line results |
| PTC Therapeutics, Inc. | PTCT | Sephience (sepiapterin) | Phenylketonuria | Q4 2026 — PTC completed the ST-920 acquisition and expects to complete the rolling BLA submission for accelerated approval in Q4 2026. |
| Scholar Rock Holding Corp | SRRK | apitegromab | Spinal muscular atrophy (SMA), non-ambulatory Type 2/3 | H2 2026 — Topline data from Phase 1 healthy-volunteer study |
| Denali Therapeutics Inc. | DNLI | AVLAYAH (tividenofusp alfa-eknm; DNL310) | neurologic manifestations of Hunter syndrome (MPS II) | — |
| Dyne Therapeutics, Inc. | DYN | z-rostudirsen (DYNE-251) | Duchenne muscular dystrophy (exon 51 skipping) | January 21, 2027 — FDA action on BLA (PDUFA target date) |
| Recursion Pharmaceuticals, Inc. | RXRX | REC-4881 (MEK1/2 inhibitor) | Familial adenomatous polyposis (TUPELO) | November 2026 — Full Phase 1b/2 trial dataset for REC-4881 will be presented at the CGA-IGC Annual Meeting on November 2, 2026. |
| Pharvaris N.V. | PHVS | Deucrictibant IR capsule (on-demand) | On-demand treatment of hereditary angioedema (HAE) attacks | Q1 2027 — Topline data from Part 1 of CREAATE in AAE-C1INH |
| ADARx Pharmaceuticals, Inc. | ADRX | onvuzosiran | Prevention of hereditary angioedema (HAE) attacks | Q4 2027 — STOP-HAE Phase 3 topline data |
| Sarepta Therapeutics, Inc. | SRPT | ELEVIDYS (delandistrogene moxeparvovec-rokl) and PMO products EXONDYS 51, VYONDYS 53, AMONDYS 45 | Duchenne muscular dystrophy | H2 2026 — Further Phase 1/2 data |
| Intellia Therapeutics, Inc. | NTLA | lonvoguran ziclumeran (lonvo-z; NTLA-2002) | hereditary angioedema (HAE) | H1 2027 — US commercial launch if approved |
| Ultragenyx Pharmaceutical Inc. | RARE | Dojolvi (triheptanoin); portfolio also Crysvita, Mepsevii | long-chain fatty acid oxidation disorders (LC-FAOD) | H2 2026 — Complete enrollment in Phase 2/3 Aurora study |
| Taysha Gene Therapies, Inc. | TSHA | TSHA-102 | Rett syndrome (MECP2 loss-of-function) | H1 2027 — REVEAL pivotal 6-month interim analysis topline data and FDA regulatory feedback toward BLA submission |
| Prime Medicine, Inc. | PRME | PM577a | Wilson disease | H1 2027 — BLA submission |
| Solid Biosciences Inc. | SLDB | SGT-003 | Duchenne muscular dystrophy | Q4 2026 — FDA meeting to discuss INSPIRE DUCHENNE data package and potential accelerated approval pathway |
| Zevra Therapeutics, Inc. | ZVRA | MIPLYFFA (arimoclomol) | neurological manifestations of Niemann-Pick disease type C (NPC), in combination with miglustat | H2 2026 — Follow-up FDA meeting on regulatory options to potentially accelerate development program (after Type C meeting in 2026-Q1) |
| Monopar Therapeutics | MNPR | ALXN1840 (tiomolybdate choline) | Wilson disease | 2026 — Complete rolling NDA submission (FDA filing acceptance decision to follow) |
| REGENXBIO Inc. | RGNX | RGX-121 (clemidsogene lanparvovec) | Mucopolysaccharidosis type II (MPS II, Hunter syndrome) | Q4 2026 — Topline data from ATMOSPHERE and ASCENT pivotal trials |
| Capricor Therapeutics, Inc. | CAPR | deramiocel | Duchenne muscular dystrophy cardiomyopathy | 2026 — Final Phase 1 results |
| Benitec Biopharma Inc. | BNTC | BB-301 (AAV9 'Silence and Replace' ddRNAi gene therapy) | Oculopharyngeal muscular dystrophy (OPMD)-related dysphagia | October 2026 — Late-breaking poster of interim Phase 1b/2a results (12-month Cohort 1, interim Cohort 2) at ESGCT |
| Arcturus Therapeutics Holdings Inc. | ARCT | ARCT-032 (LUNAR-CF) | Cystic fibrosis (CFTR mRNA, inhaled) | — |
| Abeona Therapeutics Inc. | ABEO | ZEVASKYN (prademagene zamikeracel) | Wounds in recessive dystrophic epidermolysis bullosa (RDEB) | — |
| Rocket Pharmaceuticals, Inc. | RCKT | RP-A501 | Danon disease | H1 2027 — Company expects dosing completion of pivotal Phase 2 Cohort 4 for RP-A501 in Danon Disease by mid-2027. |
| Larimar Therapeutics, Inc. | LRMR | nomlabofusp | Friedreich's ataxia | 2027 — Planned commercial launch if approved |
| Protalix BioTherapeutics, Inc. | PLX | Elelyso (taliglucerase alfa) | Gaucher disease | — |
| Niagen Bioscience, Inc. | Unknown | NB4168 | Ataxia-Telangiectasia (A-T) | — |
| Korro Bio, Inc. | KRRO | KRRO-121 | hyperammonemia in urea cycle disorders and hepatic encephalopathy | H2 2026 — Commence first-in-human trial |
| PepGen Inc. | PEPG | PGN-EDODM1 | Myotonic dystrophy type 1 (DM1) | November 2026 — PepGen expects to report clinical data from the 10 mg/kg multiple-dose cohort of FREEDOM2 in November 2026. |
| Entrada Therapeutics, Inc. | TRDA | ENTR-601-44 | Duchenne muscular dystrophy (exon 44 skipping) | Q1 2027 — Cohort 2 topline |
| Sionna Therapeutics, Inc. | SION | SION-719 | Cystic fibrosis | — |
| Satellos Bioscience Inc. | MSLE | Forazapadin (SAT-3247), oral AAK1 inhibitor | Duchenne muscular dystrophy (DMD) | Q4 2026 — Start of Phase 2 trial in adults with FSHD |
| Spruce Biosciences, Inc. | SPRB | TA-ERT (tralesinidase alfa) | Mucopolysaccharidosis IIIB (Sanfilippo B) | Q4 2026 — BLA submission for accelerated approval |
| Cumberland Pharmaceuticals Inc | CPIX | ifetroban | cardiomyopathy associated with Duchenne muscular dystrophy (also SSc, IPF, cancer metastasis) | date not stated — Top-line results |
| Polaryx Therapeutics, Inc. | PLYX | PLX-200 (gemfibrozil) | Lysosomal storage disorders (CLN2, CLN3, Krabbe, Sandhoff) | H2 2026 — Initiation of SOTERIA Phase 2 proof-of-concept basket trial |
| Rafael Holdings, Inc. | RFL | Trappsol Cyclo (hydroxypropyl beta cyclodextrin) | Niemann-Pick Disease Type C1 (NPC1) | Q4 2026 — Rafael Holdings remains on track to submit an NDA for Trappsol Cyclo in the fourth quarter of 2026. |
| Eloxx Pharmaceuticals, Inc. | ELOX | exaluren | Alport syndrome with nonsense mutations (NMAS) | Q3 2027 — First patient dosing in EXPRESS Phase 2 trial of exaluren in ADPKD expected in third quarter 2027. |
| Mereo BioPharma Group plc | MREO | setrusumab (BPS-804/UX143) | osteogenesis imperfecta (OI) | — |
| Inmune Bio, Inc. | INMB | CORDStrom | Recessive Dystrophic Epidermolysis Bullosa (RDEB) | December 2026 — US BLA submission (10-K target; undated in later 10-Q) |
| Aardvark Therapeutics, Inc. | AARD | ARD-101 | Hyperphagia associated with Prader-Willi Syndrome | — |
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.