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Research through the 18 September 2026 close

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Rare disease & gene therapy theme · 23 dossiers

Dossiers in the Rare disease & gene therapy theme cluster — 23 names moving on shared catalysts.

DormantLast produced 7 August 2026, 44 days ago — outside the 14-day window the model counts as current work.

All 23 are dated research with a published kill line — the level named in advance that would prove the call wrong.

All dossiers
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… Cyclical recovery · Rare disease & gene therapy · Precision biotech & therapeutics catalyst · MGTX MeiraGTx Holdings plc fresh 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… Earnings inflection · Rare disease & gene therapy · Precision biotech & therapeutics analysed · AGIO Agios Pharmaceuticals, Inc. fresh MEDIUM Agios Pharmaceuticals' mitapivat label-expansion thesis turns on FDA approval for sickle cell disease by 2026-11-01. Approval would validate the regulatory case; a weekly close below $29 would invalidate the price structure first. Earnings inflection · Binary-catalyst biotech · Rare disease & gene therapy catalyst · BOLD Boundless Bio, Inc. fresh MEDIUM Boundless Bio’s merger thesis pairs a planned $44–48 million cash distribution with a minority Serapha interest. Completion by 2026-12-31 with that distribution intact would confirm the case; a weekly close below $2.40 would invalidate its… Earnings inflection · M&A & special situations · Rare disease & gene therapy catalyst · MNPR Monopar Therapeutics Inc. fresh 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… Earnings inflection · Binary-catalyst biotech · Rare disease & gene therapy analysed · PGEN Precigen, Inc. fresh 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… Earnings inflection · Precision biotech & therapeutics · Rare disease & gene therapy catalyst · RGEN Repligen Corporation fresh MEDIUM Repligen's bioprocessing recovery and BioLife deal support a breakout thesis above the $182.03 market high. BioLife approval on 2026-10-05 and a weekly close above that high would confirm the next leg; a weekly close below $158 would… Cyclical recovery · Rare disease & gene therapy catalyst · 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… Earnings inflection · Rare disease & gene therapy · Precision biotech & therapeutics catalyst · ARTV Artiva Biotherapeutics, Inc. fresh LOW Artiva Biotherapeutics’ AlloNK thesis hinges on expanded rheumatoid arthritis data and pivotal-trial initiation by year-end 2026. Those milestones must arrive before a weekly close below $8.61 invalidates the market case. Earnings inflection · Rare disease & gene therapy catalyst · AUTL Autolus Therapeutics plc LOW Autolus Therapeutics’ AUCATZYL growth story lost price confirmation when the 2026-09-11 close breached the published $2.05 weekly threshold. A recovery case requires a weekly reclaim above $2.05 and delivery against the $140–150 million… Defensive · Oncology & immunology · Rare disease & gene therapy analysed · CABA Cabaletta Bio, Inc. fresh 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.… Earnings inflection · Precision biotech & therapeutics · Rare disease & gene therapy analysed · CLPT ClearPoint Neuro, Inc. fresh 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… Cyclical recovery · Medtech & diagnostics · Rare disease & gene therapy catalyst · DSGN Design Therapeutics, Inc. fresh LOW Design Therapeutics’ frataxin-biomarker thesis depends on its Q4 2026 update establishing a regulatory path for DT-216P2. Supportive FDA feedback and a defined registrational design would substantiate the case; a weekly close below $13.50… Earnings inflection · Precision biotech & therapeutics · Rare disease & gene therapy analysed · DTIL Precision BioSciences, Inc. LOW Precision BioSciences’ hepatitis B gene-editing thesis needs replicated liver-biopsy results by year-end 2026. The case succeeds if additional patients show cccDNA reduction without treatment-limiting safety findings before a weekly close… Earnings inflection · Rare disease & gene therapy · Precision biotech & therapeutics analysed · EDIT Editas Medicine, Inc. fresh LOW Editas Medicine's EDIT-401 recovery thesis failed its published price test when the 2026-09-11 weekly close reached $2.70, below $2.75. The company's Q1 2027 data-update guidance leaves human lipid lowering unresolved; the market failure… Special situation · Rare disease & gene therapy analysed · LRMR Larimar Therapeutics, Inc. fresh 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… Earnings inflection · Rare disease & gene therapy · Binary-catalyst biotech catalyst · NGNE Neurogene Inc. fresh LOW Neurogene's financed Rett gene-therapy thesis turns on Embolden meeting its clinical endpoint in the second half of 2027 before a weekly close below $30 breaks the market structure. Earnings inflection · Rare disease & gene therapy analysed · QURE uniQure N.V. fresh 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… Earnings inflection · Precision biotech & therapeutics · Rare disease & gene therapy catalyst · RARE Ultragenyx Pharmaceutical Inc. fresh 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… Earnings inflection · Rare disease & gene therapy · Binary-catalyst biotech analysed · RCKT Rocket Pharmaceuticals, Inc. fresh LOW Rocket Pharmaceuticals’ Danon recovery case requires clinical progress at the 2026-10-06 webinar and a weekly recovery above $3.86 before a weekly close below $3.20. The September 15 FDA-alignment announcement narrows regulatory… Earnings inflection · Rare disease & gene therapy · Binary-catalyst biotech catalyst · RZLT Rezolute, Inc. fresh 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"… Earnings inflection · Binary-catalyst biotech · Rare disease & gene therapy analysed · SLDB Solid Biosciences Inc. LOW Solid Biosciences’ SGT-003 thesis depends on functional Duchenne data supporting an accelerated-approval pathway. Favorable disclosed FDA feedback would validate the case; an adverse regulatory outcome or a weekly close below $8.00 would… Earnings inflection · Rare disease & gene therapy · Binary-catalyst biotech catalyst · TSHA Taysha Gene Therapies, Inc. fresh 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… Earnings inflection · Rare disease & gene therapy · Precision biotech & therapeutics analysed ·
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