Stocks · Theme

Rare disease & gene therapy stocks · 30 research notes

30 Rare disease & gene therapy stocks moving on the same story, each with a dated research note: thesis, kill line and next catalyst.

Active themeStage: accelerating, as of 15 November 2026.

All 30 have a dated research note with a published kill line: the note's own test, stated in advance, of what would prove it wrong: a price level, a business fact, or both.

ETONEton PharmaceuticalsMedium Eton Pharmaceuticals is modestly more likely than not to beat the S&P 500 over three months, after third-quarter revenue of $57.9 million, up 158%, lifted its 2026 guide above $175 million. The fourth-quarter report, expected around…Rare disease & gene therapywritten 15 November 2026 · catalyst 16 November 2026 XERSXeris Biopharma HoldingsMedium Xeris Biopharma's shares are modestly more likely than not to beat the S&P 500 over three months because Recorlev sales rose 93% to $71.5 million in the third quarter and 2026 revenue guidance rose to $395–405 million. Preliminary…Rare disease & gene therapywritten 15 November 2026 · catalyst 17 November 2026 COAGHemab Therapeutics HoldingsMedium Hemab Therapeutics is modestly more likely than not to beat the S&P 500 over three months: its pivotal sutacimig trial has started and cash is guided into 2029. Updated Factor VII deficiency data at the ASH meeting on 14 December 2026 test…Rare disease & gene therapywritten 13 November 2026 · catalyst 17 November 2026 CYRXCryoPortMedium Cryoport's cold-chain business for cell and gene therapies is growing faster and posted a second positive adjusted EBITDA quarter, with 2026 revenue guidance raised to $197–199 million. The fourth-quarter report settles whether that pace…Rare disease & gene therapy · Precision biotech & therapeuticswritten 12 November 2026 · catalyst 22 November 2026 IRONDisc MedicineMedium Disc Medicine's shares are modestly favoured to beat the S&P 500 over three months because bitopertin passed its Phase 3 APOLLO trial on 19 October 2026, answering the FDA's February rejection. ASH pipeline data from 12 December 2026 and…Rare disease & gene therapy · Binary-catalyst biotechwritten 15 November 2026 · catalyst 12 December 2026 LQDALiquidiaMedium Liquidia Corporation's shares are modestly more likely to beat the S&P 500 because YUTREPIA sales rose 31% to $222.7 million in the third quarter with record October shipments; preliminary fourth-quarter sales, due in early January 2027,…Cardiovascular therapeutics · Rare disease & gene therapywritten 15 November 2026 · catalyst 17 November 2026 PVLAPalvella TherapeuticsMedium Palvella Therapeutics has pulled back from its record close while the FDA reviews QTORIN rapamycin for a 28 February 2027 decision, with no adverse review news and $236.7 million in cash; review updates and the fourth-quarter Phase 3 start…Rare disease & gene therapywritten 12 November 2026 · catalyst 8 December 2026 VRDNViridian TherapeuticsMedium Viridian Therapeutics' shares are modestly more likely than not to beat the S&P 500: Lumvoa's $24.7 million first full quarter beat estimates and VRDN-008 data on 9 November 2026 added an autoimmune program. Fourth-quarter Lumvoa sales,…Rare disease & gene therapywritten 15 November 2026 · catalyst 16 November 2026 CAPRCapricor TherapeuticsLow Capricor Therapeutics' shares are more likely to lag the S&P 500 because deramiocel's approval faces an FDA panel that voted 9-3 against its evidence in July 2026. The FDA's action on the narrowed application, due by 22 November 2026,…Rare disease & gene therapywritten 12 November 2026 · catalyst 22 November 2026 CRSPCRISPR TherapeuticsLow CRISPR Therapeutics shares are more likely to lag the S&P 500 over three months: they hit a 52-week closing low of $42.62 on 12 November 2026 despite strong CTX310 data, and December's zugo-cel data at ASH is the next test.Rare disease & gene therapy · Cardiovascular therapeuticswritten 13 November 2026 · catalyst 17 November 2026 SEPNSepternaLow Septerna's shares are more likely to lag the S&P 500 over the next three months after a third-quarter 2026 revenue and loss miss on 9 November 2026; SEP-479 Phase 1 liver-safety data, due in the first quarter of 2027, settle the case.Rare disease & gene therapywritten 13 November 2026 · catalyst 16 November 2026 LRMRLarimar TherapeuticsMedium Larimar Therapeutics is modestly more likely than not to lead the S&P 500 over three months: it finished its nomlabofusp filing on 9 November 2026 and seven insiders bought shares on 11 November 2026 and 12 November 2026. The FDA's filing…Rare disease & gene therapy · Binary-catalyst biotechwritten 13 November 2026 RZLTRezoluteMedium Rezolute's ersodetug rests on the tumor hyperinsulinism upLIFT study, with topline guided to mid-December 2026; the final responder rate against the 7-of-8 interim settles it, and a $400 million registration filed 5 November 2026 raises…Binary-catalyst biotech · Rare disease & gene therapywritten 11 November 2026 TVTXTravere TherapeuticsMedium Travere Therapeutics' FILSPARI kidney-drug sales beat the third-quarter benchmark at $200.2 million, but new patient start forms slipped to 1,932 and Q4 carries an inventory drag. Fourth-quarter sales and the 1 December CEO handover decide…Precision biotech & therapeutics · Rare disease & gene therapywritten 10 November 2026 · catalyst 1 December 2026 ABEOAbeona TherapeuticsLow Abeona Therapeutics' ZEVASKYN revenue grew 18% to $13.5 million in the third quarter but missed estimates, and a $3.75-a-share placement plus a stock-funded drug purchase left the shares at $2.30; they are more likely to lag the S&P 500.Rare disease & gene therapy · Precision biotech & therapeuticswritten 12 November 2026 AGIOAgios PharmaceuticalsLow Agios Pharmaceuticals' shares are more likely to lag the S&P 500 until the FDA rules on mitapivat in sickle cell disease by 1 February 2027; thalassemia sales are growing, but new prescriptions are slowing and a safety program is possible.Binary-catalyst biotech · Rare disease & gene therapywritten 12 November 2026 · catalyst 12 December 2026 ARCTArcturus Therapeutics HoldingsLow Arcturus Therapeutics has cash into 2028 but no dated FDA path for ARCT-810 and no dated Phase 3 decision for ARCT-032, so the shares are more likely to lag the S&P 500 until the company sets those dates.Precision biotech & therapeutics · Rare disease & gene therapywritten 12 November 2026 CLPTClearPoint NeuroLow ClearPoint Neuro is more likely to lag the S&P 500 over three months: its fortunes ride on uniQure's Huntington's application, and its own 2026 revenue guide has been cut twice. The full-year report and the FDA's 1 May 2027 target date…Medtech, diagnostics & life-science tools · Rare disease & gene therapywritten 13 November 2026 · catalyst 31 December 2026 CPIXCumberland PharmaceuticalsLow Cumberland Pharmaceuticals shares are more likely to lag the S&P 500 while the ifetroban NDA for Duchenne heart disease has no date; $80.6 million of cash at 30 September 2026 settles funding, and a dated FDA submission would settle the…Rare disease & gene therapy · Precision biotech & therapeuticswritten 12 November 2026 DSGNDesign TherapeuticsLow Design Therapeutics' shares are more likely to lag the S&P 500: a new $200 million stock-sale program adds supply while DT-216P2's regulatory path is unconfirmed; the company's Q4 2026 registrational-plans update settles it.Precision biotech & therapeutics · Rare disease & gene therapywritten 12 November 2026 DTILPrecision BioSciencesLow Precision BioSciences' hepatitis B gene-editing data held up in full, but with no clinical result due before Phase 2 dosing in early 2027 the shares are more likely to lag the S&P 500. Year-end 2026 Duchenne safety data is the next dated…Rare disease & gene therapywritten 11 November 2026 MGTXMeiraGTx HoldingsLow MeiraGTx's shares are more likely to lag the S&P 500 over three months: the US filing for its gene therapy bota-vec slipped to the first quarter of 2027, and the December 2026 European submission is the next test.Rare disease & gene therapy · Precision biotech & therapeuticswritten 13 November 2026 MNPRMonopar TherapeuticsLow Monopar Therapeutics' Wilson disease drug ALXN1840 keeps an open FDA path, but the shares are more likely to lag the S&P 500 over three months. Completion of the New Drug Application, guided on 12 November 2026 to within several weeks, and…Binary-catalyst biotech · Rare disease & gene therapywritten 13 November 2026 NGNENeurogeneLow Neurogene's Rett syndrome gene therapy NGN-401 is funded into the first quarter of 2029, but the shares are more likely to lag the S&P 500 over three months: they closed at $23.90 on 12 November 2026, through the kill line. The Embolden…Rare disease & gene therapywritten 13 November 2026 · catalyst 31 December 2026 PGENPrecigenLow Precigen's PAPZIMEOS is now profitable and cash-generating, but quarterly growth slowed to 37% and a rival RRP drug was approved, so the shares are more likely to lag the S&P 500 over three months; fourth-quarter revenue settles it.Rare disease & gene therapy · Precision biotech & therapeuticswritten 12 November 2026 QUREuniQureLow uniQure's Huntington's gene therapy AMT-130 cleared the FDA filing stage with priority review and a 1 May 2027 decision date, yet the shares still closed at $22.71 on 10 November 2026; the advisory committee decides whether the weakened…Rare disease & gene therapy · Binary-catalyst biotechwritten 11 November 2026 RCKTRocket PharmaceuticalsLow Rocket Pharmaceuticals' shares are more likely to lag the S&P 500 over three months: the Danon gene therapy trial reports no efficacy until mid-2028, and first KRESLADI patient onboarding, guided for the fourth quarter of 2026, is the next…Rare disease & gene therapy · Binary-catalyst biotechwritten 13 November 2026 SLDBSolid BiosciencesLow Solid Biosciences' Duchenne gene therapy SGT-003 is more likely to lag the S&P 500 over three months: the Food and Drug Administration (FDA) Type C meeting in late 2026 and its minutes decide whether accelerated approval stays open.Rare disease & gene therapy · Binary-catalyst biotechwritten 13 November 2026 · catalyst 16 November 2026 SRPTSarepta TherapeuticsLow Sarepta Therapeutics is more likely to lag the S&P 500 over three months: ELEVIDYS sales fell to $83.5 million in the third quarter of 2026 from $98.1 million, and the fourth quarter is guided flat. The RNA data guided for late 2026 are…Rare disease & gene therapywritten 13 November 2026 · catalyst 28 February 2027 TSHATaysha Gene TherapiesLow Taysha Gene Therapies' Rett syndrome therapy TSHA-102 is more likely to lag the S&P 500 over three months; pivotal six-month interim data and FDA feedback on the filing route, guided to the first half of 2027, settle it.Rare disease & gene therapy · Precision biotech & therapeuticswritten 14 November 2026