Filtered library · Theme
Rare disease & gene therapy theme · 45 dossiers
Dossiers in the Rare disease & gene therapy theme cluster — 45 names moving on shared catalysts.
DormantLast produced 7 August 2026, 34 days ago — outside the 14-day window the model counts as current work.
All 45 are dated research with a published kill line — the level named in advance that would prove the call wrong.
All dossiers TVTX Travere Therapeutics, Inc. HIGH The FSGS/IgAN ramp story stalled rather than broke: the 2026-09-04 close of $65.28 sits 3.3% under the 2026-08-27 high of $67.52 with RSI(14) decayed from 65.6 to 56.0 and no published target change since Piper Sandler's $68 on 2026-08-17.… CYRX CryoPort, Inc. MEDIUM Q2 (2026-08-06) split the read: revenue $49.0M beat $48.21M and FY26 guide affirmed at $192–196M with adjusted EBITDA crossing to +$0.4M, but commercial CGT growth halved to +9% YoY from +26%. The narrative leg is now the profitability… ETON Eton Pharmaceuticals, Inc. MEDIUM The post-print re-rate topped at an all-time closing high of $64.43 on 2026-08-24 and faded 7.2% to $59.78 by 2026-08-28, with no broker revision since 2026-08-14 and a second Form 4 disposal (director, 2026-08-28) after the CEO's 100,000… MGNX MacroGenics, Inc. MEDIUM MGC026's head-and-neck cohort cleared its pre-specified Stage 1 threshold on 2026-07-23 and the interim Phase 1 data lands as ESMO poster 1020P on 2026-10-23; the name walks in with $327M pro forma cash (2026-06-30) built from the Bora… SLDB Solid Biosciences Inc. MEDIUM Post-Sarepta DMD vacuum trade still in its waiting phase, with the sell-side moving before the company does: no release since the 2026-08-06 Q2 print, but Truist carried Buy/$20 on 2026-08-21 and Cantor raised to $26 on 2026-08-27. The… DTIL Precision BioSciences, Inc. LOW First clinical evidence that a gene editor removes hepatitis-B cccDNA (EASL late-breaker, 2026-05-27, <1% remaining in one biopsy) is the whole story; the shares closed $7.47 on 2026-09-04, 19.5% under the $9.28 52-week high, with no… EDIT Editas Medicine, Inc. LOW Legacy pivot on one asset: after halting ex vivo reni-cel in December 2024, Editas is EDIT-401 — an in vivo LNP editor for HeFH showing ~90%+ NHP reductions in LDL-C, Lp(a) and ApoB, with an Australian CTN submitted in August 2026 and four… SLN Silence Therapeutics Plc LOW First-in-class siRNA re-rated on the 2026-08-10 SANRECO readout (88% response vs 19% placebo, n=48) and immediately funded with a ~$175M raise priced at $13.50/ADS; with Phase 3 not starting until 1H 2027, the equity now trades on… CRNX Crinetics Pharmaceuticals, Inc. SUPREME The settlement claim resolved: the Vertex merger became effective 2026-09-01 at $85.00 a share in cash, Nasdaq trading was suspended before that day's open and a Form 25 was requested. The final print is $84.95 on 2026-08-31; shares are… KALV KalVista Pharmaceuticals, Inc. SUPREME Resolved cash takeout, not a live security: Chiesi's $27.00/share acquisition closed 2026-06-11 and KALV stopped trading on Nasdaq that morning. An EDGAR check on 2026-09-05 shows no issuer filing since the 2026-08-14 Schedule 13G/A… PVLA Palvella Therapeutics, Inc. HIGH The undated FDA window closed on 2026-08-31: the rolling NDA for QTORIN rapamycin in microcystic lymphatic malformations is complete under 505(b)(2), starting a 60-day agency clock on filing acceptance and the Priority Review decision that… AGIO Agios Pharmaceuticals, Inc. MEDIUM Mitapivat sickle-cell label-expansion binary into the 2026-11-01 PDUFA, funded by a real ramp (Q2 mitapivat revenue $44.7M vs $26.03M consensus, 2026-07-30). Nothing company-dated has landed since; the tape closed $33.10 on 2026-09-04… BOLD Boundless Bio, Inc. MEDIUM Merger-arb wrapper on a cash shell: a planned $44–48M pre-close cash dividend plus a 3.69% stub in the AATD base-editing NewCo. Eleven weeks after the 2026-06-23 announcement the Form S-4 is still absent — EDGAR's latest filing of any kind… DSGN Design Therapeutics, Inc. MEDIUM Frataxin-protein-surrogate leg: RESTORE-FA's primary endpoint is now blood FXN protein change, and Larimar's FDA alignment on skin FXN as a surrogate for accelerated approval gives that path an external precedent. The company has issued… DYN Dyne Therapeutics, Inc. MEDIUM Nothing company-dated until the ~late-October Q3 print, leaving a maturing narrative to trade on small-cap biotech flow ahead of the 2027-01-21 PDUFA. KNSA Kiniksa Pharmaceuticals International, plc MEDIUM Post-print commercial re-rating has stopped receiving new information: the $980–995M FY26 ARCALYST guidance raise is 33 days old, no sell-side target has moved since 2026-07-30, and the 2026-08-12 fireside produced no number. Price closed… MESO Mesoblast Limited MEDIUM FY2026 printed 2026-08-27 — revenue US$120.3M vs US$17.2M, net loss down 44% to US$57.5M, cash US$103M — and the ADR still closed $17.32 on 2026-09-04, capped by the 2026-07-15 high of $18.07. The catalyst the July leg leaned on has… MGTX MeiraGTx Holdings plc MEDIUM The $400M Oberland royalty deal (2026-07-07) took dilution and runway risk off the table and spiked MGTX to a new 52-week high of $15.35 before a fade to ~$12.44 — a non-dilutive re-rate of a three-program AAV gene-therapy pipeline, but… MIRM Mirum Pharmaceuticals, Inc. MEDIUM Rare-disease franchise ran to a $125 ATH on FOP data, then cooled ~9% into a confirmed 2026-07-30 Q2 print. Base compounds (Q1 sales +43% YoY, FY guide $660–680M) with FOP PDUFA 2026-09-26 and HDV Phase 3 topline 2H26 stacked ahead — but… MNPR Monopar Therapeutics Inc. MEDIUM The rolling-NDA leg rebuilt without news: $107.72 on 2026-08-21 to $117.56 on 2026-09-04, back within 5.1% of the $123.84 high, on no company release since 2026-08-19 and with two directors selling into $109–$113 on 2026-08-25 and… NGNE Neurogene Inc. MEDIUM Rett gene-therapy story stays fully financed (~$360.2M pro forma, runway into Q1 2029) and clean at 1E15 vg across N=35 at the 2026-08-10 cutoff. The 2026-09-09 IRSF community webinar is the only company-dated appearance before the… PGEN Precigen, Inc. MEDIUM PAPZIMEOS launch-ramp durability is the binary — the first FDA-approved RRP therapy printed $21.6M net product revenue in Q1 2026, and the now-confirmed Aug 4 Q2 print (announced Jul 21) grades whether the ramp compounds sequentially or… PTGX Protagonist Therapeutics, Inc. MEDIUM De-risked dual-royalty story accelerating into a binary: icotrokinra (oral IL-23, J&J) already approved and paying 6–10% royalties; rusfertide (PV, Takeda) into a Q3 2026 PDUFA. Stock at a fresh ATH ($143.29, Jul 20) as the PV theme runs —… RCKT Rocket Pharmaceuticals, Inc. MEDIUM Price has stalled where the 2026-08-03 safety leg left it — $3.86 on 2026-09-04, unchanged from the 2026-08-28 close — with no company disclosure since the 08-10 Q2 print and three Form 144 sale notices filed 08-18. Two webcast fireside… RGEN Repligen Corporation MEDIUM Bioprocessing-recovery rerating now carries a deal overhang: the 2026-07-28 beat-and-raise (adj EPS $0.54 vs $0.45; FY26 sales to $813–835M) took RGEN within 3.2% of its $182.03 high, but the BioLife merger S-4 went effective and the… STTK Shattuck Labs, Inc. MEDIUM Receptor-side TL1A: SL-325 delivered the first human data for a DR3-blocking antibody on 2026-06-08 — complete receptor occupancy at 0.1 mg/kg, anti-drug antibodies in 3.7% of dosed subjects — and the shares are up 74.1% over three months.… ZBIO Zenas BioPharma, Inc. MEDIUM First-in-disease B-cell modulator: obexelimab BLA (IgG4-RD, 56% flare cut, HR 0.44) hit the FDA 2026-05-28, and the ~60-day filing determination — acceptance plus a possible Priority Review grant — is due within days, a fresh near-term… ABEO Abeona Therapeutics Inc. LOW The post-print drift has continued without repair: the 2026-08-28 close of $5.92 sits two cents above the $5.90 shelf the prior note named as the break, with RSI(14) at 21.8 and no Abeona press release at all between 2026-08-13 and… AUTL Autolus Therapeutics plc LOW The post-raise re-rating has stalled. After the 2026-08-03 guidance raise to $140–150M and the 2026-08-11 Q2 beat ($45.7M vs $34.0M consensus), the shares set a $2.54 52-week high and then closed $2.24 on 2026-09-04 with RSI(14) back at… CABA Cabaletta Bio, Inc. LOW Autoimmune CD19 CAR-T reset story, now funded — $225.1M cash at 2026-06-30, runway into mid-2027 — but registrational RESET-Myositis data are guided to mid-2027 and the BLA to 2H27, so no clinical datapoint resolves inside 30 days.… CLDX Celldex Therapeutics, Inc. LOW Single-asset binary: the Phase 3 EMBARQ-CSU1/CSU2 topline guided to September/October 2026 is the entire equity, and the window opens within days. The pre-readout re-rating is built and cooling — $39.12 on 2026-08-28, 12.8% under the… CLPT ClearPoint Neuro, Inc. LOW Picks-and-shovels delivery play on CNS gene therapy: ClearPoint's SmartFlow cannula and MRI navigation are the standard route to the brain for uniQure's AMT-130 Huntington's BLA (soft Q3-2026 window). The June re-rate held near $18.72… CPIX Cumberland Pharmaceuticals Inc. LOW Deal-arb thesis is now spent: the $100M Apotex asset sale closed 2026-07-01 after a 99.84% shareholder vote on 2026-06-24, so the dated binary that drove the ~3.2x off $1.85 to a $7.36 high is consumed. What remains is a cash-rich clinical… DNTH Dianthus Therapeutics, Inc. LOW The August re-rate has rolled over on no news: the last Dianthus disclosure is the 2026-08-04 Q2 print, and the 2026-08-28 close of $105.84 sits 8.7% under the $115.96 high with RSI(14) at 41.9 versus 56.6 a week earlier. Nothing… LEGN Legend Biotech Corporation LOW In-vivo CAR-T re-rate has matured into digestion: EHA (6/13) confirmed LB2501's 100% ORR and clean durability, but the 6/17 $226M raise at $29.35 capped the leg and JPM's 7/13 Neutral $35 reinstatement caps the Street. No fresh catalyst… LRMR Larimar Therapeutics, Inc. LOW Single-asset FA regulatory binary sitting on the line that defines it: the week ending 2026-09-04 closed $3.83, 0.8% above the twice-tested $3.80 shelf, after five weeks with no company press release since the 2026-08-04 Q2 update. The… MAZE Maze Therapeutics, Inc. LOW APOL1 kidney proof-of-concept intact and funded into 2029, but the only news since the last note was a conference calendar: the 2026-09-02 release scheduled fireside chats on Sept 9, 10 and 15 and repeated the late-2026/early-2027 HORIZON… MBX MBX Biosciences, Inc. LOW The Q3 commitment landed early — first patient dosed in the pivotal Phase 3 oPTimize trial on 2026-08-27 — and the tape sold it: $66.33 on 08-21 to $61.72 on 09-04, RSI(14) 30.2, 12.7% under the $70.70 high. The compiled consensus stayed… QURE uniQure N.V. LOW FDA's 2026-06-17 reversal put AMT-130 on an accelerated-approval path with a Q3 2026 BLA — the narrative is genuinely repaired, but the move is spent: $40.25 (7/17) sits below the $45.50 June offering price with the CEO selling into it on… RARE Ultragenyx Pharmaceutical Inc. LOW The +50% June reset rally round-tripped from ~$33.46 to $28.62 (2026-07-21), parking price back on the breakout shelf ahead of two binary FDA decisions: DTX401 (GSD Ia) PDUFA Aug 23 and UX111 (Sanfilippo A) PDUFA Sep 19. The July 30 Q2… RZLT Rezolute, Inc. LOW The August screener bid fully round-tripped: $5.09 on 2026-08-21 to $4.58 on 2026-09-04, back under the $4.60/$4.61 summer shelf, with no clinical news since 2026-06-17. The registrational upLIFT topline is still guided only to "2H-2026"… SANA Sana Biotechnology, Inc. LOW Type-1-diabetes functional-cure narrative: UP421's no-immunosuppression islet transplant is still producing insulin at 14 months (n=1), de-risking SC451's IND guided "as early as 2026." Rare-disease cell therapy is accelerating, but the… SPRB Spruce Biosciences, Inc. LOW Ultra-rare ERT filing story cooling: after the 2026-08-24 pre-BLA clearance the news flow stopped, and the shares drifted from the 2026-08-28 close of $60.78 to $57.93 on 2026-09-04 with RSI(14) back to 58.1 from 77.6. Nothing… TSHA Taysha Gene Therapies, Inc. LOW De-risking is behind the stock and the calendar in front of it is empty: after the clean 2026-06-22 data and the $6.00 June raise, no company-dated event lands before the ~November Q3 update, and the tape has drifted to a $5.60 close on… ZVRA Zevra Therapeutics, Inc. LOW The June patent re-rating fully reversed: EMA's CHMP issued a negative opinion on arimoclomol's EU application 2026-07-24 (efficacy not proven), and ZVRA gapped ~24% to the high-$9s, cutting through the $11.50 base. EU expansion is now…