Filtered library · Theme
rare-disease-gene-therapy theme · 29 dossiers
Dossiers in the rare-disease-gene-therapy theme cluster — 29 names moving on shared catalysts.
All dossiersMIRM
Mirum Pharmaceuticals, Inc.
Rare-disease platform re-rating on stacked de-risking: zilurgisertib FOP PDUFA set for 2026-09-26 after a 99.9% HO-lesion reduction, brelovitug HDV Phase 3 topline due 2H26, and Q1 sales +43% YoY with 2026 guide lifted to $660–680M. Six…
AGIO
Agios Pharmaceuticals, Inc.
Mitapivat SCD label-expansion re-rate: positive RISE UP Phase 3 (Jun 15) + FDA Priority Review acceptance (Jul 7, ~+15%) + same-day PT cluster to $46. The binary payoff is the ~Nov 1 2026 PDUFA; the acceptance pop is already digested and…
DSGN
Design Therapeutics, Inc.
Post-print collapse fully reversed: DSGN re-rated +40% in June to $15.19 (2026-07-10), reclaiming the $14.37 data-day high and pressing toward the $17.25 52-week high as the FA gene-therapy theme accelerated and the pipeline broadened…
MAZE
Maze Therapeutics, Inc.
APOL1 kidney precision-medicine story re-accelerating post-HORIZON: Phase 2 (Mar-25-2026) gave first broad-AMKD proof-of-concept (35.6% proteinuria cut, 61.8% in FSGS), stock reclaimed the April $23.50 raise, and a second oral asset…
MGTX
MeiraGTx Holdings plc
MGTX defended the $9.00 April-raise base on 2026-06-05, then reclaimed the $11.85 prior high to print a new 52-week high of $12.11 (2026-06-26). Price is leading the news a base-hold breakout bought ahead of far-dated 2027 binaries (AQUAx2…
NGNE
Neurogene Inc.
NGN-401 Rett-syndrome gene-therapy narrative re-accelerated on the 2026-06-29 low-dose data update, strong enough to place a $125M raise at $30 the next day. Data and financing are now both out, so the next binary is FDA…
PGEN
Precigen, Inc.
PAPZIMEOS launch inflection accelerating the first and only FDA-approved RRP therapy printed $21.6M net product revenue in Q1 2026 (a beat), with cash-flow breakeven guided by year-end and orphan exclusivity to 2032; the ~Aug 10 Q2 print…
PVLA
Palvella Therapeutics, Inc.
First-ever-approval-for-microcystic-lymphatic-malformations narrative accelerating and de-risked: SELVA hit its endpoint (Feb 24, 2026), FDA granted rolling NDA review, first module filed June 29, 2026. But no binary inside 30 days NDA…
SLDB
Solid Biosciences Inc.
Next-gen DMD gene therapy filling the vacuum from Sarepta Elevidys's safety collapse; SGT-003 clean across 40 dosed (no DILI/TMA/myocarditis, May 4 2026 cutoff) with ~110% microdystrophin expression. +172% off the low to $10.42 the…
TSHA
Taysha Gene Therapies, Inc.
Rett gene-therapy story de-risked: clean REVEAL Part A 12-month data (100% response across N=12, deepening effect, zero treatment-related SAEs; June 22 2026) plus the pivotal fully dosed and a $200M raise at $6.00 funding the BLA path. The…
TVTX
Travere Therapeutics, Inc.
FILSPARI FSGS launch ramp is re-rating into all-time highs near $58: a third June analyst-target wave (Citi $70, Guggenheim $65, Wedbush $61, Evercore $60) is marking up the dual-indication ramp. But the stock now trades inside that target…
URGN
UroGen Pharma Ltd.
ZUSDURI launch ramp still accelerating Q1'26 revenue $51.0M (+152% YoY), ZUSDURI $29.2M (+109% QoQ), prescribers 102→256 and the stock has now broken out above the old $32.37 cap to a fresh 52-week high ($39.59). Spot sits above the…
VIR
Vir Biotechnology, Inc.
Post-fade base reclaim is live: shares pushed from ~$8.86 (Jun 17) back above the 50-DMA to ~$10 on roughly 5x average volume, with 11% short interest as fuel. The partnered Astellas/VIR-5500 TCE story is funded into H2 2028; the next hard…
ZVRA
Zevra Therapeutics, Inc.
MIPLYFFA's patent cliff was pushed to 2041 (Orange Book listing 2026-06-08), and three weeks on the re-rating has held ZVRA sits ~$13.80, near its $14.58 52-week high, rather than fading. But the discrete catalyst is behind the tape; the…
BHVN
Biohaven Ltd.
Spec-biotech de-rate reversal on opakalim epilepsy data, now >110% off the $7.48 low to ~$16; RISE3 enrollment complete and shares shrugged off a BofA Underperform ($11), but the value-driving topline is unscheduled 2H-26 and a dilution…
CPIX
Cumberland 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…
CYRX
CryoPort, Inc.
CGT cold-chain picks-and-shovels; the 2026 re-rate has matured. Consensus PT drifted to $15.11 (now just below the ~$15.26 spot), the rating softened to Moderate Buy with two holds added, and insiders distributed into the early-June $16.73…
ETON
Eton Pharmaceuticals, Inc.
Q1 beat-and-raise (adj EPS $0.14 vs $0.09, FY26 guide lifted above $120M, 2026-05-14) confirmed the specialty-pharma rollup, but the story stopped accelerating in mid-May and now drifts through an empty window into the ~August Q2 print the…
HROW
Harrow, Inc.
Commercial ophthalmology special-situation trading on one number: does the guided H2 ramp print. June–July portfolio launches (VERKAZIA, BYOOVIZ) keep sentiment alive, but the binary is the ~Aug 10 Q2 report $71–81M guide vs $44.2M Q1. A…
KALV
KalVista Pharmaceuticals, Inc.
The Chiesi takeout is done: the tender expired with ~77.8% of shares in (43,152,532 sh), the second-step merger went effective 2026-06-11 at $27.00/share cash, and KALV ceased trading on Nasdaq before the open the same day. No US-listed…
LEGN
Legend Biotech Corporation
In-vivo CAR-T re-rate has resolved into digestion: EHA full data (6/13) confirmed LB2501's 100% ORR and durable persistence, but the 6/17 $226M raise at $29.35 capped the move and the stock now sits at the offering price. Maturing…
LRMR
Larimar Therapeutics, Inc.
Single-asset FA binary: nomlabofusp rolling BLA underway for accelerated approval on a novel frataxin surrogate, Breakthrough-designated and FDA-aligned but shares sit near 52-week lows after a -15% sell-the-news on the 2026-06-29 filing,…
NKTR
Nektar Therapeutics
Feb-2026 REZOLVE-AD maintenance pop to $109 has fully round-tripped; NKTR has stabilized near $62 — but the next true binary (ZENITH-AD Phase 3 AD readout) is mid-2028, so there is no accelerating leg to buy. The mechanism is de-risked yet…
OMER
Omeros Corporation
The EU upside binary broke: CHMP issued a negative opinion on YARTEMLEA in TA-TMA (2026-06-26), gapping OMER ~20% to ~$8.70 and slicing the $8.50 base. What remains is a US-only orphan launch (Q1 net $9.9M, Q2 consensus ~$11.6M) plus a…
QURE
uniQure N.V.
Real FDA U-turn (AMT-130 BLA Q3) but binary post-5x into a just-priced $225M dilution; already hold immunology via KYMR.
RARE
Ultragenyx Pharmaceutical Inc.
Post-setrusumab-failure reset is now a cleaner two-shot gene-therapy story: DTX401 (GSD Ia) PDUFA ~Aug 23 and UX111 (Sanfilippo A) PDUFA ~Sep 19 are paired binaries, with Angelman GTX-102 Phase 3 topline due H2 2026. Stock ran ~50% into…
RCKT
Rocket Pharmaceuticals, Inc.
Post-crash gene-therapy rebuild: first commercial approval (KRESLADI/LAD-I) plus a non-dilutive $180M PRV sale (closed 2026-06-12) fund it into Q2 2028, but the real re-rate is the undated H2 2026 RP-A501 Danon update a binary on a program…
SANA
Sana 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…
DYN
freshDyne Therapeutics, Inc.