Upfront Briefing
The FDA issued a CRL for REGENXBIO’s Hunter syndrome gene therapy — a reminder that “ultra-rare + external controls + biomarker surrogates” still faces real regulatory friction at the finish line.
On the deal tape, Lilly kept writing checks (up to ~$2.4B for Orna; plus a $350M-upfront Innovent pipeline pact with milestone back-end), while AgomAb’s $200M IPO closed and multiple private rounds printed across the AI/drug discovery stack.
Tape Action
| Instrument |
Last close |
1D % |
YTD % |
| S&P 500 |
6,964.8 |
+0.5% |
+1.7% |
| Nasdaq 100 |
25,268.1 |
+0.8% |
+0.1% |
| Russell 2000 |
2,689.1 |
+0.7% |
+8.3% |
| Healthcare (XLV) |
156.3 |
(0.9%) |
+1.0% |
| Biotech (XBI) |
126.1 |
+0.5% |
+3.4% |
| Nasdaq Biotech (NBI) |
5,970.1 |
(0.2%) |
+4.6% |
| Clinical Trials (BBC) |
41.0 |
+0.2% |
+6.2% |
|
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Broad tape finished green with mega-cap/growth leadership (Nasdaq-100 outperformed).
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Healthcare (XLV) lagged: a sharp drawdown in lab tools (notably Waters after guidance) weighed on the sector even as the index tape stayed risk-on.
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Biotech internals were mixed (XBI up modestly while NBI dipped), consistent with stock-picking rather than a clean beta day.
The Big 3
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1
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FDA rejects Regenxbio gene therapy for Hunter syndrome
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The FDA issued a Complete Response Letter (CRL) for REGENXBIO’s RGX-121 (Hunter syndrome/MPS II), citing concerns around trial design and eligibility criteria, the comparability of the external natural-history control, and whether the CSF biomarker surrogate is reasonably likely to predict clinical benefit.
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Why it matters:
This is another reminder that “ultra-rare + accelerated approval + external controls” is not a free pass—FDA is tightening the bar on surrogate endpoints and comparability. Practically, a CRL can force longer follow-up or additional study work, extending time-to-revenue and raising read-through risk for other CNS/lysosomal gene therapy programs leaning on biomarker endpoints.
- Source: STAT
- More: Endpoints; Reuters
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2
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Lilly to buy Orna in ~$2.4B platform deal (in vivo CAR-T bet)
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Eli Lilly agreed to acquire Orna Therapeutics in a deal reported at up to ~$2.4B, adding an RNA/LNP-enabled platform aimed at generating CAR-T cells in vivo (with early focus on autoimmune indications).
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Why it matters:
Big-cap willingness to pay real money for pre-/early-platform optionality is the signal. This sets a valuation reference point for in vivo cell engineering and RNA delivery platforms—and can re-rate adjacent private comps (plus pull forward BD interest for “platform + wedge program” stories).
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Source:
PR
- More: Reuters
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| 3 | QuantX Biosciences raises $85M in Series B financing |
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QuantX Biosciences closed an oversubscribed $85M Series B. Syndicate: co-led by Lilly Asia Ventures (LAV) and Sanofi Ventures; joined by Hongshan plus existing investors.
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Why it matters:
A “real” mid-cycle Series B with pharma venture participation is a useful risk-appetite read—especially when proceeds are earmarked to push named programs toward the clinic. It can also pull forward partnering expectations (or set a higher bar for platform stories that can’t show near-term IND cadence).
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Source:
PR
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Everything Else that broke
- RFK Jr. comments on European drug prices and tariffs. — Endpoints
- VolitionRx receives NYSE American non-compliance notice. — PR
- FDA grants MUMS status for AB Science's Masivet in canine tumors. — PR
- MerryLife reports positive Phase 1 TML-6 results in Alzheimer's. — PR
- Applied Medical wins $381M antitrust verdict against Medtronic. — PR
- Ferronova completes enrollment for gastric/oesophageal cancer trial. — PR
Deal Flow
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BioBucks 2026 Deal Trackers • Updated weekly
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M&A / BD&L
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Lilly to buy Orna in deal worth up to ~$2.4B (cash; incl. upfront + milestones) to add an in vivo CAR-T/RNA platform. —
Reuters
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Iambic–Takeda AI discovery partnership: upfront/research/tech-access payments + success-based payments potentially >$1.7B, plus royalties. —
Business Wire
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XOMA completes acquisition of Generation Bio: $4.2913/share cash + a non-tradeable CVR. —
PR
VC / Private Financings
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QuantX closes oversubscribed $85M Series B — co-led by Lilly Asia Ventures (LAV) + Sanofi Ventures; joined by Hongshan + existing investors. —
PR
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Galux raises $29M Series B — joined by InterVest + DAYLI Partners + PATHWAY Investment (existing); plus Yuanta + Korea Development Bank (KDB) + SL Investment (new) (and others). —
PR
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Aerska Therapeutics raises $39M Series A — led by EQT Life Sciences Dementia Fund + age1; joined by Iaso Ventures + existing investors. —
PR
IPOs / Follow-Ons
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SpyGlass Pharma closes $172.5M IPO with full overallotment. —
PR
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Satellos closes US$57.2M public offering (incl. overallotment). —
PR
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AgomAb closes $200M IPO (12.5M ADS @ $16; underwriters option 1.875M ADS). —
PR
Academic Corner
- Sensitive detection of cancer antigens enabled by user-defined peptide libraries. — Nature Biotech
- Reliability of LLMs as medical assistants for the general public: a randomized preregistered study. — Nature Medicine
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