Filtered library · Theme
Rare disease & gene therapy theme · 35 dossiers
Dossiers in the Rare disease & gene therapy theme cluster — 35 names moving on shared catalysts.
All dossiers AGIO Agios Pharmaceuticals, Inc. MEDIUM Mitapivat SCD label-expansion binary into a 2026-11-01 PDUFA, now with a real commercial leg under it: Q2 mitapivat revenue $44.7M vs $26.03M consensus (2026-07-30), yet the tape gave the beat back — $33.56 close on 2026-08-14, RSI 35.8,… AKTS Aktis Oncology, Inc. MEDIUM The "coverage is spent" read broke: Wedbush ($35) and BMO ($37) initiated Outperform on 2026-08-19, taking the cohort to five, and the 2026-08-13 Q2 reaffirmed Q1 2027 AKY-1189 data with $517.3M cash. Price did not follow — $24.07 on… 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… DNTH Dianthus Therapeutics, Inc. MEDIUM Complement re-rate has gone quiet: no company disclosure since the 2026-08-04 Q2 print, a fresh 52-week high at $115.96 on a lower RSI than the early-August one, and the next dated binary — MoMeNtum MMN top-line — not until December 2026.… DYN Dyne Therapeutics, Inc. MEDIUM maturing — nothing company-dated until the ~late-October Q3 print, then the 2027-01-21 PDUFA. KNSA Kiniksa Pharmaceuticals International, plc MEDIUM Post-print commercial re-rating still working but no longer fed by new information: the 2026-07-28 FY26 ARCALYST guidance raise to $980–995M and the 07-29/07-30 target ladder are three weeks old, the 08-12 Canaccord fireside produced no… LRMR Larimar Therapeutics, Inc. MEDIUM Single-asset FA binary, now basing rather than breaking: the 2026-08-04 Q2 update held anaphylaxis at 10 of 43, reported no new urticaria after antihistamine premedication, and put cash at $156.3M with runway into Q3 2027. Rolling BLA… MBX MBX Biosciences, Inc. MEDIUM Single-asset execution story: oPTimize Phase 3 in canvuparatide is guided to start by 2026-09-30 and sites are already screening. Since 2026-08-15 the sell-side re-rated on no new data — consensus $88.36 across 12 analysts (2026-08-23) vs… 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… NGNE Neurogene Inc. MEDIUM Rett gene-therapy story is now fully financed — pro forma cash ~$360.2M after the July raise, runway into Q1 2029 — and the 2026-08-11 Q2 print added safety breadth (N=35 at 1E15 vg as of 08-10) but zero new efficacy. Next hard binary is… 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 —… PVLA Palvella Therapeutics, Inc. MEDIUM First-approved-therapy-for-microcystic-LM story still working: the 2026-08-04 Q2 update reaffirmed rolling-NDA completion in H2 2026 and potential approval H1 2027, and the stock closed $156.13 on 2026-08-14, 1.0% under its 52-week high.… RCKT Rocket Pharmaceuticals, Inc. MEDIUM First clean RP-A501 datapoint in 15 months — three Danon patients dosed at the recalibrated 3.8×10¹³ GC/kg with no TMA or capillary leak (2026-08-03) — turned a bleeding chart into a mid-range one. The re-rate still needs the undated H2… TVTX Travere Therapeutics, Inc. MEDIUM Q2 beat on 2026-08-04 ($169.6M revenue vs $159.6M consensus; FILSPARI US sales +96% YoY to $141.1M) re-opened sell-side headroom — six target raises 08-05→08-10 into a $70–85 band while the 2026-08-14 close of $62.14 sits 1.0% under the… 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 2026-08-13 Q2 print missed both lines — revenue $11.38M vs ~$12.24M, EPS -$0.35 vs -$0.23 — and price has given back 18.4% from the $7.46 high. But treated patients rose to five from three and the QTC network hit its seven-center 2026… BOLD Boundless Bio, Inc. LOW Merger-arb wrapper, not an oncology stock: the object is a planned $44–48M pre-close cash dividend plus a 3.69% stub in the AATD base-editing NewCo. Week of 2026-08-14 added owners (ADAR1 13G at 8.8%) but no deal documents — still no S-4,… 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… CRNX Crinetics Pharmaceuticals, Inc. LOW Not a narrative any more: the Vertex $85.00/share cash takeout cleared HSR on 2026-08-12, the stockholder vote is 2026-08-28, and closing is guided to early September. At the 2026-08-14 close of $84.70 the gap to terms is $0.30 against… DSGN Design Therapeutics, Inc. LOW A new 52-week high of $15.94 printed in August with no company release since the 2026-08-03 Q2 update, which pushed the next hard data to Q1 2027. RSI(14) 73.9 at the $14.99 close (2026-08-21) and +21.9% for the month, into a 30-day… KALV KalVista Pharmaceuticals, Inc. LOW Resolved cash takeout, not a live security: Chiesi's $27.00/share acquisition closed 2026-06-11, KALV delisted the same morning, Form 15-12G filed 2026-06-22, and Rule 12g-4 registration termination lands ~2026-09-20. The 2026-06-10 close… 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… MAZE Maze Therapeutics, Inc. LOW APOL1 kidney precision-medicine story intact, but the 2026-08-11 Q2 print emptied 2026 of catalysts: MZE782 PKU topline pushed to 2027 and CKD Phase 2 start to H1 2027, leaving only HORIZON's three 10–15 patient cohorts in… MESO Mesoblast Limited LOW Platform-inflection story re-accelerated on operational news rather than data: the 2026-08-17 last-patient-treated release for the 350-patient CLBP Phase 3 carried the ADR from $15.60 (08-14) to $17.00 (08-21), reclaiming the July shelf… MNPR Monopar Therapeutics Inc. LOW New 52-week high at $123.84 printed and sold — the 2026-08-21 close was $107.72, 13.0% under it, RSI 46.0, on no company news. The rolling NDA clock started 2026-07-22 is live but still undated, and the sell-side finished repricing in… 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 A 10.7% week to $5.09 on 2026-08-21 with zero company news since the 2026-07-21 conference notice — the bid arrived via Friday-gainer screens, not a disclosure. The 2026-06-02 upLIFT interim (6 of 8 responders) is still the entire story,… 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… SLDB Solid Biosciences Inc. LOW Post-Sarepta DMD vacuum trade now in a waiting phase: the guided mid-2026 accelerated-approval update never landed — the 2026-08-06 Q2 release moved the FDA meeting to late Q4 2026 while INSPIRE dosing grew to 53 (as of 2026-08-04). Price… TSHA Taysha Gene Therapies, Inc. LOW Clinical binary printed clean (2026-06-22: 100% milestone response N=12, zero treatment-related SAEs) but the next catalyst slipped to H1 2027 — pivotal six-month interim AND FDA BLA-pathway feedback both guided to the first half of next… VIR Vir Biotechnology, Inc. LOW Q2 (2026-08-05) printed $238.9M revenue and $0.47 diluted EPS on Astellas upfront recognition — an accounting swing, and the tape read it as one: $9.24 on 2026-08-14, still under the $10.36 Astellas equity strike, with Morgan Stanley and… 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…