Nasdaq
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We are a clinical-stage biopharmaceutical company pioneering the discovery and development of regulatory RNA-based therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Our principal executive offices are located in Cambridge, Massachusetts.
Roles derived from SEC Form 4 filings.
Actuals from SEC filings; YoY vs the year-ago quarter. Analyst estimates & surprise require a data feed (coming soon).
| Line item | FY2023 | FY2024 | FY2025 |
|---|---|---|---|
| Revenue | $350,000.00 | $652,000.00 | $3.5M |
| Gross profit | — | — | — |
| Operating income | -$51.9M | -$53.1M | -$52.6M |
| Net income | -$49.3M | -$51.8M | -$80.4M |
| Diluted EPS | -$124.80 | -$11.04 | -$2.65 |
| Operating cash flow | -$44.2M | -$45.6M | -$29.6M |
| Free cash flow | -$44.8M | -$46.0M | -$29.8M |
| Gross margin | — | — | — |
| Operating margin | -14822.6% | -8142.3% | -1502.4% |
| Net margin | -14083.1% | -7943.4% | -2298.5% |
| Line item | Q2 '24 | Q3 '24 | Q4 '24 | Q1 '25 | Q2 '25 | Q3 '25 | Q4 '25 | Q1 '26 |
|---|---|---|---|---|---|---|---|---|
| Revenue | $0.00 | $0.00 | $652,000.00 | $858,000.00 | $1.5M | $795,000.00 | $348,000.00 | $1.3M |
| Gross profit | — | — | — | — | — | — | — | — |
| Operating income | -$12.7M | -$13.5M | -$14.0M | -$13.1M | -$13.0M | -$13.7M | -$12.8M | -$13.1M |
| Net income | -$12.6M | -$13.5M | -$13.3M | -$12.4M | -$12.6M | -$15.1M | -$40.3M | -$18.3M |
| Diluted EPS | -$26.00 | -$24.19 | $65.72 | -$0.62 | -$0.62 | -$0.55 | -$0.86 | -$0.32 |
| Operating cash flow | -$13.4M | -$9.7M | -$11.3M | -$14.3M | -$10.3M | -$11.2M | $6.3M | -$11.0M |
| Free cash flow | -$13.6M | -$9.7M | -$11.6M | -$14.6M | -$10.3M | -$11.2M | $6.3M | -$11.0M |
| Gross margin | — | — | — | — | — | — | — | — |
| Operating margin | — | — | -2152.6% | -1526.8% | -870.3% | -1717.1% | -3671.3% | -1010.1% |
| Net margin | — | — | -2036.7% | -1449.1% | -840.8% | -1899.2% | -11575.9% | -1416.6% |
Consensus revenue & EPS estimates and price targets will appear here once a forward-estimates data feed is connected.
| Date | Insider | Type | Shares | Price | Value | Filing |
|---|---|---|---|---|---|---|
| 2025-09-11 | Schwab Andrew J. | Award | 2,941,176 | $1.53 | $4.5M | filing |
| 2025-09-11 | 5AM Partners VI, LLC | Buy | 2,941,176 | $1.53 | $4.5M | filing |
| 2025-09-11 | Nashat Amir | Buy | 1,221,721 | $1.53 | $1.9M | filing |
| 2025-09-11 | Polaris Management Co. VII, L.L.C. | Buy | 1,221,721 | $1.53 | $1.9M | filing |
| 2025-09-11 | Nashat Amir | Buy | 85,468 | $1.53 | $130,766.04 | filing |
| 2025-09-11 | Polaris Management Co. VII, L.L.C. | Buy | 85,468 | $1.53 | $130,766.04 | filing |
| 2025-09-11 | Young Richard A | Buy | 15,151 | $1.65 | $24,999.15 | filing |
| 2025-09-11 | Maricich Yuri· Chief Medical Officer | Buy | 6,060 | $1.65 | $9,999.00 | filing |
| 2025-09-11 | Gold Kelly· Chief Financial Officer | Buy | 6,060 | $1.65 | $9,999.00 | filing |
| 2025-09-11 | Mandel-Brehm Josh· Chief Executive Officer | Buy | 6,060 | $1.65 | $9,999.00 | filing |
Latest 10 transactions.
Fundamentals & filings from SEC EDGAR. Prices adjusted for splits & dividends. Not investment advice.