Benitec Biopharma (BNTC)
Develops BB-301, a ddRNAi genetic medicine for OPMD dysphagia.
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Develops genetic medicines using ddRNAi and a Silence and Replace approach, with BB-301 as the lead OPMD program. The business remains pre-commercial and centered on U.S.-based clinical execution, regulatory progress, and the durability of its gene-silencing platform.
Builds genetic medicines from ddRNAi, a platform that uses gene-therapy delivery to sustain RNA-interference activity. The current pipeline is centered on BB-301, a Silence and Replace candidate for OPMD-related dysphagia that aims to silence mutant PABPN1 while supplying a functional replacement gene.
Operations are concentrated in the United States, with a Hayward, California headquarters and a small team weighted toward research and development. Recent opened company updates describe continued BB-301 patient follow-up, high-dose cohort treatment, and no treatment-related severe adverse events.
FDA Fast Track status and orphan-drug designations from FDA and EMA support the regulatory pathway, while the business remains dependent on clinical results, regulatory design, and competition from larger gene-therapy and gene-silencing organizations.
Company facts
Target rangeAs of Sep 2, 2026
Latest analyst notes
| Date | Firm | Action | Rating | Prior target | Target |
|---|---|---|---|---|---|
| Jul 28, 2026 | Wedbush | Initiate | Outperform | $16 | |
| May 12, 2026 | Evercore ISI | Target | $25 | ||
| Nov 24, 2025 | H.C. Wainwright | Target | $35 | $32 | |
| Nov 4, 2025 | H.C. Wainwright | Target | $35 | ||
| Nov 4, 2025 | JMP Securities | Target | $18 | $22 |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Mar 31, 2026Cash & short-term investments $184.8M · Total debt $876.0K · Total assets $187.0M · Shareholders' equity $183.3M
- Revenue
- $0
- Operating income
- −$13.6M
- Net income
- −$11.9M
No dividends on record.
| Institutions88 holders · as of Mar 31, 2026 | 94.55% | 32.48M shares | |
| Insiders2 holders · as of Jun 30, 2026 | 0.47% | 161.7K shares | |
| Otherremainder | 4.98% | 1.71M shares |
34.35M company shares as of Sep 3, 2026 · institutions as of Mar 31, 2026 · holders as of their latest filing
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Evidence |
|---|---|---|---|
Suvretta Capital Management, LLCInstitution | 33.62% | 11.55M | 13Fas of Mar 31, 2026 |
Janus Henderson Group PLCInstitution | 8.37% | 2.88M | 13Fas of Mar 31, 2026 |
Franklin Resources IncInstitution | 6.92% | 2.38M | 13Fas of Mar 31, 2026 |
Adage Capital Partners GP, L.L.C.Institution | 6.17% | 2.12M | 13Fas of Mar 31, 2026 |
Ra Capital Management, L.P.Institution | 5.39% | 1.85M | 13Fas of Mar 31, 2026 |
| See all 25 → | |||
13F — a manager's quarterly holdings
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Dec 23, 2025 | Suvretta Capital Management, LLC | director, 10 percent owner: | +50,000 | $12.85 |
| Dec 26, 2025 | Suvretta Capital Management, LLC | director, 10 percent owner: | +16,836 | $13.10 |
| Dec 29, 2025 | Suvretta Capital Management, LLC | director, 10 percent owner: | +77,387 | $13.44 |
| Dec 18, 2025 | Suvretta Capital Management, LLC | director, 10 percent owner: | +10,215 | $11.24 |
| Dec 19, 2025 | Suvretta Capital Management, LLC | director, 10 percent owner: | +16,384 | $11.48 |
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Compare
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Benitec Biopharma | $12.88 | +4.63% | $442.5M | — | — | 795.8 | — |
| Sarepta Therapeutics | $23.15 | −0.26% | $2.45B | — | 28.9 | 1.2 | −34.3% |
| Arrowhead Pharmaceuticals | $85.13 | −0.30% | $12.02B | — | — | 18.0 | +171.0% |
| Dyne Therapeutics | $25.14 | +0.16% | $4.69B | — | — | — | — |
| Novartis (ADR) | $161.87 | −0.35% | $308.79B | 24.5 | 17.3 | 5.6 | +0.9% |
| Alnylam Pharmaceuticals | $265.22 | −0.74% | $35.78B | 45.2 | 31.5 | 7.4 | +66.9% |
| uniQure | $46.16 | −0.17% | $3.21B | — | — | 171.7 | +11.0% |
| REGENXBIO | $9.60 | +3.00% | $496.3M | — | — | 2.8 | +405.7% |
| Rocket Pharmaceuticals | $3.85 | −0.26% | $421.5M | — | — | — | — |
| Median | 34.8 | 28.9 | 6.5 | +38.9% |










