Rare Disease— Companies & Signals
The rare disease investment theme centers on companies developing therapies for conditions affecting small patient populations, addressing significant unmet medical needs. This focus offers the potential for substantial returns due to premium pricing, extended market exclusivity, and expedited regulatory pathways. 170 public companies above the $50M floor. Last 90 days: 89 red 8-Ks, 0 insider cluster-buys, 12 running hot in the press.
170
Companies
89
Red 8-Ks · 90d
0
Insider clusters
12
🔥 Media-hot
Companies in Rare Disease
Refine in search →| # | Ticker | Company | Sector | Mkt cap | 1D | 5D | Volume | Heat | Signals |
|---|---|---|---|---|---|---|---|---|---|
| 1 | XFOR | X4 Pharmaceuticals, Inc | Healthcare | $377.3M | +5.2% | +2.7% | 592K | — | red 8-K |
| 2 | IMMX | Immix Biopharma, Inc. | Healthcare | $601.6M | +5.1% | +11.6% | 1.9M | — | |
| 3 | TVTX | Travere Therapeutics, Inc. | Healthcare | $4.8B | -0.9% | +5.8% | 1.4M | ⚡ | red 8-K |
| 4 | ARCT | Arcturus Therapeutics Holdings Inc. | Healthcare | $198.1M | -0.9% | -5.7% | 395K | — | red 8-K |
| 5 | AAPG | ASCENTAGE PHARMA GROUP INTERNATIONAL | Healthcare | $1.6B | +5.5% | +3.3% | 11K | — | |
| 6 | MIRM | Mirum Pharmaceuticals, Inc. | Healthcare | $6.1B | -0.6% | +14.1% | 964K | — | |
| 7 | ZVRA | ZEVRA THERAPEUTICS, INC. | Healthcare | $747.8M | +3.6% | +10.8% | 1.5M | — | |
| 8 | BBIO | BridgeBio Pharma, Inc. | Healthcare | $13.1B | +2.5% | +4.5% | 3.3M | — | red 8-K |
| 9 | RLMD | RELMADA THERAPEUTICS, INC. | Healthcare | $697.5M | +4.3% | +1.3% | 2.3M | — | |
| 10 | ANIP | ANI PHARMACEUTICALS INC | Healthcare | $1.9B | -0.5% | +4.4% | 316K | — | red 8-K |
| 11 | IONS | IONIS PHARMACEUTICALS INC | Healthcare | $12.1B | -0.6% | +6.3% | 2.8M | — | red 8-K |
| 12 | PLYX | Polaryx Therapeutics, Inc. | Healthcare | $118.1M | +5.4% | +5.0% | 337K | — | |
| 13 | SLGL | Sol-Gel Technologies Ltd. | Healthcare | $240.1M | +1.2% | +0.8% | 15K | — | |
| 14 | ANTX | AN2 Therapeutics, Inc. | Healthcare | $170.7M | -3.3% | -6.8% | 324K | — | |
| 15 | RNAC | Cartesian Therapeutics, Inc. | Healthcare | $279.4M | +2.3% | -3.1% | 192K | — | red 8-K |
| 16 | CAPR | CAPRICOR THERAPEUTICS, INC. | Healthcare | $1.5B | -10.0% | -18.9% | 3.2M | — | |
| 17 | SRPT | Sarepta Therapeutics, Inc. | Healthcare | $1.6B | +1.0% | -3.6% | 3.8M | — | red 8-K |
| 18 | INSM | INSMED Inc | Healthcare | $21.2B | +1.3% | +6.2% | 2.7M | — | |
| 19 | ABEO | ABEONA THERAPEUTICS INC. | Healthcare | $315.2M | +0.9% | -1.6% | 1.3M | — | red 8-K |
| 20 | LRMR | Larimar Therapeutics, Inc. | Healthcare | $360.5M | -12.6% | -12.6% | 13.4M | — | |
| 21 | RFL | Rafael Holdings, Inc. | Healthcare | $116.5M | +14.5% | +14.9% | 333K | — | |
| 22 | CRDL | Cardiol Therapeutics Inc. | Healthcare | $118.7M | -1.0% | -2.8% | 342K | — | |
| 23 | VNDA | Vanda Pharmaceuticals Inc. | Healthcare | $353.0M | +2.2% | +2.3% | 874K | — | red 8-K |
| 24 | AGIO | AGIOS PHARMACEUTICALS, INC. | Healthcare | $1.8B | +0.4% | +8.4% | 772K | — | red 8-K |
| 25 | CHRS | Coherus Oncology, Inc. | Healthcare | $215.9M | -3.3% | -2.0% | 1.1M | — | red 8-K |
| 26 | CLLS | Cellectis S.A. | Healthcare | $310.9M | +3.9% | +4.3% | 22K | — | |
| 27 | IRWD | IRONWOOD PHARMACEUTICALS INC | Healthcare | $600.8M | +4.1% | +12.6% | 3.5M | — | red 8-K |
| 28 | ZNTL | Zentalis Pharmaceuticals, Inc. | Healthcare | $266.9M | +14.1% | +25.6% | 2.8M | — | red 8-K |
| 29 | RLYB | Rallybio Corp | Healthcare | $92.3M | +0.4% | +0.8% | 31K | — | red 8-K |
| 30 | PLX | Protalix BioTherapeutics, Inc. | Healthcare | $168.4M | +2.2% | +8.8% | 541K | — | |
| 31 | TARA | Protara Therapeutics, Inc. | Healthcare | $219.2M | +1.0% | +1.2% | 989K | — | red 8-K |
| 32 | WVE | Wave Life Sciences Ltd. | Healthcare | $1.1B | -2.6% | -7.9% | 2.7M | — | |
| 33 | AVXL | ANAVEX LIFE SCIENCES CORP. | Healthcare | $231.7M | -0.8% | +2.4% | 797K | — | red 8-K |
| 34 | MGTX | MeiraGTx Holdings plc | Healthcare | $888.1M | +3.5% | +9.2% | 683K | — | |
| 35 | CRNX | Crinetics Pharmaceuticals, Inc. | Healthcare | $3.6B | +0.7% | +4.4% | 884K | — | red 8-K |
| 36 | BNTC | Benitec Biopharma Inc. | Healthcare | $384.1M | +2.7% | +3.3% | 98K | — | red 8-K |
| 37 | HRMY | Harmony Biosciences Holdings, Inc. | Healthcare | $2.0B | -0.4% | +5.7% | 830K | — | red 8-K |
| 38 | TIL | Instil Bio, Inc. | Healthcare | $53.7M | +1.4% | -0.4% | 17K | — | red 8-K |
| 39 | RCUS | Arcus Biosciences, Inc. | Healthcare | $3.0B | +0.3% | +14.1% | 1.6M | — | |
| 40 | XERS | Xeris Biopharma Holdings, Inc. | Healthcare | $1.2B | +0.7% | +12.2% | 2.6M | — | red 8-K |
| 41 | DCTH | DELCATH SYSTEMS, INC. | Healthcare | $404.6M | +0.1% | +12.5% | 641K | — | red 8-K |
| 42 | DWTX | Dogwood Therapeutics, Inc. | Healthcare | $51.8M | +7.9% | +15.6% | 56K | — | |
| 43 | KZIA | KAZIA THERAPEUTICS LTD | Healthcare | $163.2M | +1.7% | +3.3% | 195K | — | |
| 44 | MAIA | MAIA Biotechnology, Inc. | Healthcare | $79.0M | +5.0% | +7.3% | 772K | — | red 8-K |
| 45 | ALNY | ALNYLAM PHARMACEUTICALS, INC. | Healthcare | $37.8B | +0.7% | +2.5% | 1.4M | — | red 8-K |
| 46 | RYTM | RHYTHM PHARMACEUTICALS, INC. | Healthcare | $6.1B | +0.2% | +8.5% | 828K | ⚡ | red 8-K |
| 47 | GANX | Gain Therapeutics, Inc. | Healthcare | $71.7M | +13.1% | +7.0% | 12.5M | — | |
| 48 | LXRX | LEXICON PHARMACEUTICALS, INC. | Healthcare | $852.9M | +3.5% | +22.8% | 4.6M | — | |
| 49 | SRRK | Scholar Rock Holding Corp | Healthcare | $5.3B | +0.4% | +11.7% | 2.1M | — | |
| 50 | CPIX | CUMBERLAND PHARMACEUTICALS INC | Healthcare | $83.9M | +1.3% | +22.8% | 50K | — | |
| 51 | IRD | Opus Genetics, Inc. | Healthcare | $333.6M | +1.5% | -0.5% | 824K | — | red 8-K |
| 52 | CABA | Cabaletta Bio, Inc. | Healthcare | $490.8M | +2.3% | +3.3% | 5.5M | — | |
| 53 | TNXP | Tonix Pharmaceuticals Holding Corp. | Healthcare | $180.9M | -3.6% | +4.5% | 526K | — | red 8-K |
| 54 | PGEN | PRECIGEN, INC. | Healthcare | $1.6B | +1.0% | +10.0% | 6.3M | — | red 8-K |
| 55 | VSTM | Verastem, Inc. | Healthcare | $360.3M | +1.1% | -10.1% | 2.7M | — | |
| 56 | PTCT | PTC THERAPEUTICS, INC. | Healthcare | $6.2B | -3.4% | +0.8% | 4.3M | — | red 8-K |
| 57 | EWTX | Edgewise Therapeutics, Inc. | Healthcare | $3.7B | -1.3% | +12.1% | 1.9M | ⚡ | |
| 58 | OSTX | OS Therapies Inc | Healthcare | $75.7M | +19.1% | +23.1% | 2.0M | — | |
| 59 | UNCY | Unicycive Therapeutics, Inc. | Healthcare | $198.6M | +8.0% | -2.2% | 2.6M | — | red 8-K |
| 60 | INBX | Inhibrx Biosciences, Inc. | Healthcare | $1.4B | +0.7% | +2.4% | 411K | — |
Membership is matched by keyword from companies’ SEC business descriptions (10-K Item 1) — deterministic, not AI inference. May not be exhaustive; not investment advice.