Filtered library · Theme
rare-disease-gene-therapy theme · 39 dossiers
Dossiers in the rare-disease-gene-therapy theme cluster — 39 names moving on shared catalysts.
All dossiersTXG
fresh10x Genomics, Inc.
Atera's order book turned the name: the 2026-08-06 beat-and-raise first sold to $45.60, then reversed to a record $52.03 close on 2026-08-07 (+14.10%, 5.43M shares) with the 16-analyst average target moving $41.54→$46.31 in two sessions.…
AGIO
Agios Pharmaceuticals, Inc.
Mitapivat SCD label-expansion re-rate: positive RISE UP Phase 3 (2026-06-15) plus FDA Priority Review (2026-07-07) set a 2026-11-01 PDUFA binary. The acceptance pop is digested and backup asset tebapivat's 2026-07-21 Phase 2 failure thins…
CGEM
Cullinan Therapeutics, Inc.
CD19 T-cell-engager-in-autoimmune narrative re-rated CGEM ~3x off the $5.68 low on CLN-978 lupus/RA single-dose remissions; now drifting to ~$17.10 in a bull flag under the $19.43 high with the big data catalyst spent and no dated readout…
CYRX
freshCryoPort, Inc.
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
freshDianthus Therapeutics, Inc.
Complement re-rate has extended to a new 52-week high of $112.64; the 2026-08-04 Q2 print added a December MMN top-line and a new bifunctional candidate, and four broker PT raises in four sessions (08-03→08-06) are sell-side catch-up to a…
DYN
freshDyne Therapeutics, Inc.
Filing binary resolved: FDA accepted the z-rostudirsen exon-51 DMD BLA with Priority Review on 2026-07-20, PDUFA 2027-01-21 — Stock closed $26.25 on 2026-08-07 at its 52-week high with no dated catalyst for five months. MATURING: the…
ETON
freshEton Pharmaceuticals, Inc.
Rare-disease specialty-pharma rollup went from single-broker to a coverage cluster — Canaccord initiated Buy/$60 on 2026-07-17, Craig-Hallum raised to $62 on 2026-07-28 — alongside +50.4% over three months and two pipeline headlines in…
KNSA
freshKiniksa Pharmaceuticals International, plc
Rare-disease commercial re-rating: ARCALYST Q2 revenue $243.6M (+~55% YoY) beat $225.5M consensus and FY26 guidance jumped to $980–995M from $930–945M on 2026-07-28, triggering a target ladder to $90–107. The leg is now momentum with no…
MGTX
MeiraGTx Holdings plc
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.
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.
Rett gene-therapy story materially de-risked: FDA Breakthrough Therapy (Feb 27), Embolden pivotal fully dosed (Jun 8, 25 pts), clean 1E15 safety, and a $125M raise at $30 funding past 2028. But the next hard binary — Embolden topline — is…
PTGX
Protagonist Therapeutics, Inc.
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 —…
ABEO
freshAbeona Therapeutics Inc.
CMS NTAP for ZEVASKYN granted 2026-08-04 plus a confirmed 2026-08-13 Q2 date have carried ABEO to within 0.7% of its $7.29 52-week high. The launch-ramp narrative is accelerating on reimbursement news, but the print is the binary:…
AKTS
freshAktis Oncology, Inc.
Coverage-and-flow leg has crested: -25% from the 2026-06-30 all-time closing high of $32.23 to $24.13 on 2026-08-07, no new sell-side action since JPM's 2026-07-14 maintain at $33, and three insiders selling in July's post-lock-up window.…
BHVN
Biohaven Ltd.
De-rate reversal off the $7.48 Nov-CRL low has stalled near $14 after peaking ~$16 in early July; the opakalim epilepsy pivotal (RISE3) topline is unscheduled 2H-26, sell-side is turning cautious (BofA Underperform $11), and a dilution…
BOLD
freshBoundless Bio, Inc.
Still a merger-arb wrapper rather than 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. The 2026-08-07 10-Q re-documented it ($72.6M cash, 75% RIF), but price has…
CLPT
ClearPoint Neuro, Inc.
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…
CORT
freshCorcept Therapeutics Incorporated
Lifyorli oncology launch (approved 2026-03-25, NCCN preferred) re-accelerated the tape to fresh 52-week highs near $92, and the 2026-06-17 Cushing's relacorilant resubmission (~Dec PDUFA) adds second-approval optionality. But a fresh entry…
CPIX
freshCumberland Pharmaceuticals Inc.
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.
Momentum leg is dead: the story crystallized into Vertex's $85.00/share all-cash takeout (announced 2026-07-06, ~$10B). Shares gapped to ~$83.50 and now trade as a merger-arb spread capped at $85 with ~$40 of air below on a break. Residual…
DSGN
freshDesign Therapeutics, Inc.
The June–July re-rate has run into an empty calendar. The 2026-08-03 Q2 update pushed DT-168 FECD data to 2027 and re-cut RESTORE-FA to a 10-patient 12-week cohort with blood FXN protein as the primary efficacy endpoint, moving the next…
ELVN
freshEnliven Therapeutics, Inc.
June 11 was the last new clinical datapoint; what carried price to $60.46 on 2026-08-07 was the 8/5 Q2 print ($895.2M cash, runway into 2030, Fast Track) plus a same-week target cluster (Stifel $82, GS $80, Guggenheim cut to $78). Funded…
HROW
Harrow, Inc.
Commercial ophthalmology special situation trading on one number: whether the guided Q2 ramp ($71–81M vs $44.2M Q1) prints ~2026-08-11. A ~40% recovery off the May low into ASRS clinical data and a fresh BTIG $63 reiteration has front-run…
KALV
KalVista Pharmaceuticals, Inc.
Closed takeout, not a security: Chiesi's $27.00/share all-cash acquisition of KalVista completed 2026-06-11, shares delisted from Nasdaq with deregistration (POS AM, Form 25, Form 15) underway. No spread, no momentum leg, no listed share…
LEGN
Legend Biotech Corporation
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.
Single-asset FA binary: nomlabofusp's rolling BLA for accelerated approval is live, but the 2026-06-29 open-label data carried an anaphylaxis signal (10 of 43 patients, ~23%) that turned the filing into a ~17% selloff. Shares drift near…
MAZE
Maze Therapeutics, Inc.
APOL1 kidney precision-medicine story with clinical proof-of-concept in hand (Mar-25 HORIZON: 35.6% proteinuria cut, 61.8% in FSGS), but the tape has rolled over — $27.68, down 26% YTD and ~48% off the 52-wk high, no hard binary until…
MBX
MBX Biosciences, Inc.
Once-weekly canvuparatide's 1-yr Phase 2 durability (57% responders, 12 Jun) plus a cleared End-of-Phase-2 FDA path drove a +580% YTD re-rating into all-time highs. With the data catalyst spent, no dated binary inside 30 days, and an…
MNPR
Monopar Therapeutics Inc.
De-risked Wilson-disease NDA melt-up extended rather than sold the EAN news: a 6.70M-share float ran to fresh all-time highs (~$111 on 2026-07-17) on the June-30 FDA Rare Pediatric Disease designation and clustered analyst PT hikes. The…
OMER
Omeros Corporation
Post-CHMP-rejection repair, not a new leg: shares have clawed back from the 2026-06-26 gap low of $8.67 to roughly $9.69 on the J1289 J-code going live 2026-07-01. The 2026-08-13 Q2 print is the whole trade — consensus ~$11.6M net sales…
RARE
Ultragenyx Pharmaceutical Inc.
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…
RCKT
Rocket Pharmaceuticals, Inc.
Broken gene-therapy rebuild bleeding toward its 52-week floor while waiting on binaries: KRESLADI (LAD-I) approved with a Q4 2026 launch and a closed $180M PRV sale fund it, but the re-rate is the undated H2 2026 RP-A501 Danon update — a…
RZLT
Rezolute, Inc.
Post-interim re-rating has fully decayed: $4.61 on 2026-07-17 vs the $5.14 late-June high, with no dated event between here and the upLIFT full topline in 2H-2026. A binary rare-disease name in its dead zone — the 6/8 responder signal is…
SANA
freshSana Biotechnology, Inc.
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.
Post-Sarepta DMD gene-therapy vacuum trade: SGT-003 clean across 40 dosed (~110% microdystrophin) vs Elevidys's liver-death collapse. Ran ~+200% to a $11.45 high, then gave back ~29% into the $8 shelf; the overdue mid-2026 INSPIRE data +…
TSHA
Taysha Gene Therapies, Inc.
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…
TVTX
Travere Therapeutics, Inc.
FILSPARI's dual-indication ramp has fully re-rated the equity: at ~$56.71 (2026-07-17) TVTX trades at consensus PT ($52–58) and 4% under its 2026-06-25 ATH close of $59.03, having gone sideways for four weeks. The approval binary is spent;…
VIR
Vir Biotechnology, Inc.
June's volume-backed reclaim to $10.03 failed to extend — $9.43 on Jul 17, -5.3% on the week, with short interest rising to 11.72% of float as shorts pressed the fade. Structure holds above the 50-DMA near $8.71, but nothing hard is dated…
ZVRA
Zevra Therapeutics, Inc.
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…