Tenaya Therapeutics

Phase A — Understand the business

Lens 1 · Company Overview

Tenaya is a clinical-stage biotech (Delaware, inc. Aug 2016; HQ South San Francisco) with a single, disciplined mission: potentially curative therapies for the genetic drivers of heart disease. It has three clinical-stage assets, all internally discovered, plus a modality-agnostic discovery platform:

• TN-201 — AAV9 gene therapy delivering a working MYBPC3 gene for MYBPC3-associated hypertrophic cardiomyopathy (HCM). MYBPC3 mutations are the most common genetic cause of HCM, ~20% of the HCM population, >120,000 people in the U.S. Phase 1b/2 MyPEAK-1, both dose cohorts fully enrolled/dosed. Fast Track + Orphan + Rare Pediatric (FDA), Orphan (EC); EMA PRIME granted June 2026 and FDA accepted the pediatric indication into the Rare Disease Evidence Principles (RDEP) process.
• TN-401 — AAV9 gene therapy delivering a working PKP2 gene for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). PKP2 is the most common genetic cause of ARVC, >70,000 U.S. patients; sudden cardiac death is the first sign in ~25% of known cases. Phase 1b/2 RIDGE-1, both dose cohorts dosed; DSMB endorsed expansion Jan 2026. Fast Track + Orphan (FDA), Orphan (EC).
• TN-301 — oral small-molecule selective HDAC6 inhibitor for HFpEF and other cardiac/metabolic/muscular indications (genetic DCM, DMD, PAH). Phase 1 in healthy volunteers complete (clean, once-daily, dose-dependent target engagement). Not yet in patient trials. Strategy: partner it for large indications; Orphan + Rare Pediatric for DMD.

Business model. No approved products, essentially no product revenue. Value is created by advancing the pipeline through clinical milestones and either (a) commercializing the rare-disease gene therapies themselves, or (b) out-licensing the prevalent-indication small molecule. First non-dilutive validation arrived March 2026: a multi-target research collaboration with Alnylam Pharmaceuticals — Alnylam reimburses Tenaya's FTEs + out-of-pocket costs for target-validation work, pays a $10.0M upfront platform-access fee, and Tenaya is eligible for up to ~$1.1 billion in development/regulatory/sales milestones. Key contract terms are the point of the lens: the Alnylam milestones are fully constrained / optionality (recognized only when achieved) and Alnylam may unilaterally terminate for any reason on notice — so the $1.1B is a headline, not a receivable. Also holds an $8M CIRM grant (milestone-based) funding the RIDGE-1 ARVC trial.

Key stakeholders. Customers = (eventually) cardiologists/genetic-cardiomyopathy centers and payers; today the relevant "customers" are the capital markets and strategic pharma partners. Suppliers = CDMO-grade AAV inputs but Tenaya has in-house manufacturing (Union City facility). Partners = Alnylam (discovery), CIRM (grant), academic licensors (select IP).

Lens 2 · Supply Chain

For an AAV gene-therapy developer the "supply chain" is the vector-to-vein manufacturing and delivery chain plus the diagnostic funnel that finds patients. Named nodes:

Upstream (make the drug):

• AAV9 capsid + payload design — internalized: capsid engineering, novel promoters/regulatory elements, manufacturing science all in-house. This vertical integration is unusual for a company this size and is a stated moat.
• Manufacturing — Tenaya owns process/analytical development and clinical-supply manufacturing at a leased Union City, CA facility (lease to July 2031; ~$1.4M/yr rent; $8.3M undiscounted future lease payments). PP&E net $26.0M at Q1-2026, largely this plant. Owning the plant de-risks CDMO dependency but is a heavy fixed cost for a burning pre-revenue company.
• HQ / labs — South San Francisco (171 Oyster Point Blvd, ~32,370 sq ft, lease to Nov 2027).

Midstream (prove it):

• CROs run the trials; Tenaya relies on third parties for preclinical + clinical execution (a named risk-factor dependency).
• Clinical sites — leading cardiology centers in the U.S. and United Kingdom for RIDGE-1 (ARVC).

Downstream (find + treat patients):

• Genetic-testing / diagnosis funnel — the addressable patient count depends on genetic testing uptake. ACC/AHA/ESC guidelines now recommend genetic testing + family counseling; mandatory young-athlete screening is expanding the funnel. This is the real bottleneck: PKP2-ARVC "frequently goes undiagnosed."
• Seroprevalence gate — AAV9 gene therapy only works in patients without pre-existing anti-AAV9 antibodies. Tenaya runs the RIDGE natural-history/seroprevalence study; a "large majority" of PKP2-ARVC adults have low enough antibodies to be eligible. This is a genuine chokepoint for any AAV therapy and a single-point dependency on the AAV9 capsid.

Single-source / chokepoint flags: (1) AAV9 as the sole delivery vector — one capsid class carries all the gene-therapy value; (2) in-house manufacturing = concentration risk (one plant); (3) reliance on third-party CROs; (4) the immunosuppression regimen required to manage AAV-related liver-enzyme elevations (a safety chokepoint shared by the whole field — see Rocket, Lens 13).

Lens 3 · Competitive Advantages (moats)

What's genuinely defensible:

1. First-mover + clinical lead in MYBPC3-HCM. TN-201 is, on the public evidence, the most advanced gene therapy for the single largest genetic HCM population, with multi-year human biopsy/imaging data and now EMA PRIME + FDA RDEP pediatric acceptance. Competitors in HCM gene therapy (e.g., DiNAQOR) are preclinical. This is a real, if fragile, lead.
2. Vertically integrated AAV cardiac platform — capsid engineering + cardiac-tropic promoters + in-house manufacturing + iPSC/engineered-heart-tissue disease models + ML phenotypic screening. The Alnylam deal is third-party validation that this discovery know-how has standalone value.
3. Cardiac-specific know-how — dosing, biopsy, immunosuppression, and endpoint selection for heart-directed AAV is hard-won; Tenaya has run more MYBPC3/PKP2 patients than most.
4. IP + regulatory designations — exclusive worldwide rights to the lead programs; Orphan/Fast Track/Rare Pediatric/PRIME confer market exclusivity + priority-review-voucher optionality.

Bargaining power — weak where it counts. Against patients/payers Tenaya would have pricing power if approved (curative, rare, no alternatives for non-obstructive/pediatric HCM). But today the company has almost no bargaining power over its true counterparties — the capital markets — because it is sub-$1, burning, and must raise. That inverts the usual moat analysis: the science moat is real; the financial moat is nonexistent, and in biotech the financing position often dominates the outcome (see Lens 5/11/13). Switching costs / network effects are not yet relevant (no product).

Moat verdict: a legitimate scientific/first-mover moat around TN-201 specifically, thinner and contested around TN-401 (two direct competitors, Lens 13), wrapped in a balance sheet that gives the company no negotiating leverage. Moats you can't fund don't compound.

Lens 4 · Segments

No reportable product segments — single operating segment, pre-revenue. The only "segment" disaggregation that matters is R&D by activity and program, though Tenaya explicitly does not allocate R&D by program. R&D by activity, FY2025 vs FY2024:

Revenue "segment": collaboration revenue only — $0.225M in Q1-2026 (Alnylam upfront amortization + reimbursables), $0 in FY2025. Geography: U.S.-centric with U.K. clinical sites; no geographic revenue split (no revenue). Trend: every R&D line is decelerating — deliberate, via two workforce reductions (March 2025 + May 2024). This is a company shrinking spend to extend runway, not scaling for launch — a defensive posture, not offense.

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Phase B — Measure performance (clinical overlay: Lens 5 → Pipeline-by-phase; Lens 7 → Catalyst calendar + mechanism comps)

Lens 5 → Pipeline by phase (the asset table is the company)

TN-201 efficacy signal (the crux): across evaluable patients — cardiac troponin I down as much as 74% to normal/near-normal; LVPWT reduced 21–39%; LVMI down 12–22%; all patients improved ≥1 NYHA class (Cohort 1 reached NYHA I / asymptomatic); MyBP-C protein rose over time (+4% avg C1; +14% at 12wk for first C2 patient — clear dose response). Safety: no dose-limiting toxicities; the dominant AE is reversible, asymptomatic Grade 1–3 liver-enzyme elevation managed with immunosuppression, with faster tapers/lower steroid doses in C2. One patient (Pt 5) lost to follow-up after week 12. This is a genuine cardiac-remodeling signal in a curative one-time therapy — the strongest single fact in the bull case.

TN-401 efficacy signal: PVC reductions of 46% (Pt 1) and 89% (Pt 2); NSVT eliminated/stable; PKP2 protein +10% mean; no thrombotic microangiopathy, no cardiotoxicity, no ICD shocks post-treatment. Functionally strong; protein-expression modest vs the competitor (Lexeo +93–162%, Lens 13) — the open question is whether functional benefit or protein magnitude predicts durability.

Lens 6 · Earnings Calls (sentiment trend)

No transcripts on the shelf (transcripts=0); assessment from filings MD&A tone + press releases. The narrative arc has shifted from "platform build-out" to "capital discipline + regulatory alignment": two workforce reductions, R&D down 21% YoY, and repeated MD&A language that spend "will be subject to operational decisions made following data … and our ability to achieve regulatory alignment on our pivotal trial plans". Management stopped talking about breadth and now talks about funding the two lead gene therapies to a pivotal decision. The June 2026 press cadence is confident on data (remodeling, symptom relief, PRIME/RDEP) but conspicuously silent on how the pivotals get paid for — the tell of a team managing a runway problem it hasn't solved. Tone: clinically upbeat, financially guarded. (Flag: pull the Q1-2026 and Q2-2026 call transcripts on next refresh to sentiment-trend the last 3–4 calls properly.)

Lens 7 → Catalyst calendar + mechanism comps

Catalyst calendar (the value inflections):

Mechanism comps (by target, not P/E — this is pre-revenue):

Comps takeaway: TN-201 is the clear leader in its niche (a genuine white space — no approved therapy for non-obstructive or pediatric MYBPC3-HCM). TN-401 is in a contested three-horse ARVC race and appears behind Lexeo on the biomarker that Wall Street will anchor on (PKP2 protein). Traditional multiples (EV/Sales, P/E) are n/a — pre-revenue.

Lens 8 · Stock-Price Catalysts (what actually moves TNYA)

Pattern from the tape: TNYA is a clinical-milestone + financing stock, and increasingly a survival stock. 52-week range $0.53–$2.35, now ~$0.75 — i.e., it has lost the large majority of its value and sits near the low. The moves that matter: (1) clinical data readouts (AHA Nov 2025 for TN-201, HRS/ASGCT for TN-401) — but note the June 3, 2026 positive TN-201 update produced almost no reaction (+0.12% pre-news), meaning good clinical data is no longer sufficient to move the stock; (2) dilutive financings — the Dec-2025 ($55.8M) and March-2025 ($48.9M) raises, both at deep discounts with warrant sweeteners, reset the share count 2.5× and crushed the price; (3) sector-wide AAV safety events — Rocket's RP-A501 death is a read-through risk to all cardiac AAV; (4) the Nasdaq bid-price / reverse-split overhang. Revealed preference: the market is pricing balance-sheet risk far above pipeline value — it will not re-rate on data alone until the financing question is resolved.

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Phase C — Judge people & books (clinical additions: Science & exclusivity)

Lens 9 · Management

• Faraz Ali — CEO since June 2018 (founding-era CEO), director since Sept 2018. Prior: Chief Business Officer at REGENXBIO, VP Global Commercial Development at bluebird bio, roles at Sanofi Genzyme/Genzyme. HBS MBA, Stanford BS. Archetype: gene-therapy business-development operator, not a scientist-founder — exactly the profile you want for dealmaking/partnering and capital-raising, which is the company's actual bottleneck. Track record: took Tenaya public (2021 IPO after a 1:6 reverse split), built the platform, signed Alnylam. The unflattering read: he has also presided over a ~$605M accumulated-deficit burn and a stock down to $0.75 — though most of that is sector-wide gene-therapy derating, not idiosyncratic.
• Dr. Whit Tingley — CMO since Dec 2018 — cardiology/clinical leadership; the MyPEAK-1/RIDGE-1 programs are his.
• Tenure/skin in the game: management avg tenure ~3.8 yrs, board ~6.5 yrs. Insider ownership not sourced from filings here (insider-transactions.csv absent) — n/a; check the DEF 14A on refresh.
• Capital allocation: the defining decisions are (a) owning manufacturing (heavy fixed cost, strategic optionality); (b) serial dilutive equity raises at deep discounts with warrants — value-destructive to existing holders but arguably the only survival option; (c) two workforce reductions to extend runway (disciplined); (d) the Alnylam deal — genuinely good non-dilutive capital + validation. Net: a management team playing a weak hand competently, but the hand is weak.
• Red flags: no related-party or promotional-behavior flags surfaced. The honest concern is not integrity — it's whether a BD-led team can out-finance and out-execute better-capitalized competitors (Rocket, Lexeo) in ARVC and reach a self-sustaining event before dilution/delisting erodes the equity to nothing.

Lens 10 · Forensic Red Flags

Clean on the classic forensic axes; the risks are structural (dilution + going-concern-adjacent), not accounting.

• Auditor: Deloitte & Touche LLP, San Francisco, clean opinion, auditor since 2019. No going-concern qualification — but note management's own "twelve months" language is the floor, not comfort (Lens 11).
• Revenue recognition: trivially clean — the only revenue is Alnylam collaboration, single performance obligation, cost-to-cost input method, ~$10M remaining performance obligation, milestones fully constrained. No aggressive revenue.
• Cash vs earnings: FY2025 net loss $90.6M vs operating cash burn $68.3M — the $22M gap is non-cash (SBC $13.0M, D&A $8.4M). Q1-2026: net loss $19.3M vs op burn $19.2M — tight, clean, no divergence. No receivables/inventory games (no product). Q1 accounts receivable of $10.6M is the Alnylam upfront invoiced — legitimate.
• SBC: $13.0M FY2025 (down from $16.5M) — ~14% of opex; not flattering non-GAAP because Tenaya reports GAAP net loss (no adjusted-EPS games). Reasonable.
• Balance-sheet flags: no debt (SVB term loan expired, $0 outstanding; $20M available at SVB discretion). Total liabilities $28.9M at Q1-2026, mostly leases + contract liabilities. Clean.
• Dilution machine (the real "red flag"): shares 86.5M → 216.8M in FY2025 (2.5×), via 125M new units at $0.70 (March) and $1.20 (December) with attached warrants (Series A @ $0.80, Series B @ $0.70 expiring June 2026, December warrant @ $1.50). A large warrant overhang sits above the current $0.75 price — most are out-of-the-money, but they cap upside and signal how the company finances. Additional ~$69.8M ATM capacity remains. This is not fraud; it is a structural transfer of value from equity holders to new capital, and it is the dominant financial fact.

Regulatory findings (required sub-section):

• SEC Litigation Releases / AAERs: None. Verified via SEC EDGAR EFTS (LR + AAER) as of 2026-07-06 — total SEC findings = 0.
• Non-SEC enforcement (FTC/DOJ/FDA/etc.): web search surfaced no material enforcement actions, consent decrees, fines, or penalties against Tenaya Therapeutics. The only FDA interactions are favorable (Fast Track, Orphan, Rare Pediatric, RDEP acceptance).
• Item 3 Legal Proceedings (10-K, own disclosure): "We are not currently a party to any litigation or legal proceedings that are likely to have a material adverse effect on our business.".
• Conclusion: No material regulatory or legal findings — verified via SEC EDGAR EFTS (LR, AAER), web search, and 10-K Item 3 as of 2026-07-06. The Nasdaq minimum-bid-price deficiency is a listing-standard matter, not an enforcement action, but it is the live regulatory-adjacent risk (Lens 7/13).

Science & exclusivity (clinical overlay)

• Mechanism validation: MYBPC3/PKP2 are well-validated monogenic drivers — gene replacement (not editing) restores a missing structural protein; conceptually clean and de-risked vs novel targets. The human biopsy data showing DNA persistence + rising mRNA + protein expression + downstream remodeling is strong mechanistic proof for TN-201 specifically.
• KOL/publication credibility: MyPEAK-1 first-in-human results peer-reviewed in Cardiovascular Research (Oxford) — a real journal, not a press release. Cleveland Clinic commentary corroborates "safety and early efficacy".
• IP / exclusivity: exclusive worldwide rights to lead programs; Orphan Drug (7-yr US / 10-yr EU market exclusivity if approved), Fast Track, Rare Pediatric (priority-review-voucher optionality), EMA PRIME. Patent estate not detailed on shelf (patents/ empty) — n/a; the designations are the near-term exclusivity moat.
• Reimbursement path: one-time curative gene therapies for rare cardiomyopathies would command six-/seven-figure pricing with strong payer rationale (offsets lifetime HF costs/ICDs/transplant), but payer path is unproven for cardiac gene therapy and is a live commercial risk if/when approved.

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Phase D — Project & stress-test (clinical overlay: Lens 11 → rNPV + runway-to-catalyst)

Lens 11 → rNPV + Runway-to-catalyst (the lens that actually matters)

Runway is the whole ballgame. The arithmetic:

• Cash & equivalents $80.9M at March 31, 2026; zero marketable securities (all in cash).
• Q1-2026 operating burn $19.2M; FY2025 burn $68.3M (~$17M/qtr, trending down via layoffs).
• Runway ≈ $80.9M ÷ ~$18M/qtr ≈ ~4.5 quarters ≈ into H1/mid-2027. Management asserts "sufficient for at least the next twelve months" from the May-2026 filing date — i.e., through ~mid-2027. Alnylam reimbursements + the residual ATM + the $20M SVB facility (at bank discretion) extend this modestly.
• The problem: a pivotal cardiac gene-therapy program costs far more than the remaining runway. Tenaya can reach pivotal-design alignment (H2 2026) and durability data on today's cash, but it cannot fund the pivotal trials themselves without a large raise or a partner. So the base case requires a financing/partnership event in the next ~12 months, almost certainly dilutive at a depressed price — unless a pivotal-alignment or partnership print re-rates the stock first.

rNPV framing (illustrative, every input labeled ): TN-201 (MYBPC3-HCM) — peak US/EU sales in a >120k-patient orphan population at gene-therapy pricing could plausibly be **$1–2B **; at a lead-asset Phase-1b/2 PoS of **~30–40% ** and a high biotech discount rate (~14%), risk-adjusted value is a **multiple of the current ~$80M enterprise value ** — i.e., on fundamentals the equity looks cheap if it survives dilution. TN-401 adds contested option value; TN-301 + Alnylam add platform optionality. The market cap ($160M) barely exceeds cash ($80.9M), so enterprise value (~$80M) ascribes almost nothing to three clinical programs + a validated platform + the Alnylam deal. That gap is the bull case; the reason it exists is the financing/dilution/delisting risk (the bear case). Both are true at once.

Brier forecast (binary readout, per clinical overlay — not an EPS line): TN-201 achieves FDA/EMA alignment on a pivotal/registrational trial design by 2026-12-31 — p ≈ 0.55. (Not logged via forecast.ts — --watchlist unattended run; log on promotion to a thesis.) Secondary: TNYA regains/retains Nasdaq listing compliance without a value-destructive event through 2026 — p ≈ 0.5.

Lens 12 · Bull vs Bear

Bull case. Tenaya owns the most advanced gene therapy for the largest genetic HCM population, with human data showing actual cardiac remodeling (troponin −74%, wall-thickness −21–39%, NYHA improvement) from a one-time curative therapy — the holy grail in a disease with no approved therapy for the non-obstructive/pediatric segment. Regulatory momentum is real and recent (EMA PRIME, FDA RDEP pediatric acceptance, Fast Track, Orphan, Rare Pediatric). The Alnylam deal validates the discovery platform and brings non-dilutive cash + up to $1.1B milestone optionality. At an enterprise value of ~$80M, the market is paying essentially nothing above cash for all of it — a classic left-for-dead biotech where a single de-risking print (pivotal alignment, a partnership, or a clean financing) could re-rate the equity multiples. Analysts covering it are unanimously Buy (8/8), median PT $3.00, average ~$4 vs a $0.75 print — 4–5× implied.

Bear case (2–3 permanent-impairment risks). (1) Dilution/insolvency spiral: the company cannot fund its own pivotal trials, must raise into a sub-$1 stock, and each raise (see the 2.5× share-count explosion in FY2025) transfers value away from holders — the equity can be right about the science and still go to near-zero on financing. (2) Nasdaq delisting (July 27 deadline): most-likely cure is a reverse split, a distress signal that historically compounds selling. (3) Sector safety cloud + ARVC competition: Rocket's RP-A501 patient death + FDA hold taints all cardiac AAV; in ARVC specifically, Lexeo (LX2020) is ahead on PKP2 protein expression and Rocket is also in the race — TN-401 may not be best-in-class. Pre-mortem (18 months out, thesis broke): TN-201 pivotal alignment slipped or demanded a larger/longer trial than cash allows; the company did a 1-for-N reverse split and a dilutive raise at a depressed post-split price; a competitor's ARVC data or another field-wide AAV safety event compressed sentiment; the equity is down another 60–80% despite "good" clinical data — exactly the pattern the June-3 non-reaction foreshadowed. Are multiples too high? No — there are no earnings multiples; the risk is not overvaluation but survivability. Contrarian view the market refuses to see: the market is treating TNYA as a pre-bankruptcy shell, but the combination of a peer-reviewed remodeling signal + EMA PRIME + a Big-Biotech (Alnylam) platform validation + ~$80M net cash is a plausibly acquirable/partnerable asset — the entire enterprise trades below what a pharma might pay for the platform + TN-201 alone. The disconnect is real; the catalyst to close it is financing certainty, not more data.

Lens 13 · Devil's Advocate (short-seller)

Dismantling the bull case:

• The revenue is a mirage. "Up to $1.1B Alnylam milestones" is fully constrained optionality, and Alnylam can walk for any reason. Real revenue is $0.2M/qtr of reimbursement. Anyone quoting the biobucks is selling a lottery ticket.
• The balance sheet forces value destruction. ~4–4.5 quarters of cash, $69.8M ATM primed, a stock at $0.75, and pivotal trials it can't afford. The base case is dilution at distressed prices — the FY2025 playbook (units at $0.70/$1.20 + warrants, 2.5× share count) will repeat. The equity is structurally short its own financing.
• Delisting mechanics. Below $1 since January; July 27 compliance deadline; the realistic fix is a reverse split that historically precedes further declines for cash-strapped biotechs. Index/ETF exclusion + liquidity loss compound it.
• TN-401 may not be best-in-class. Lexeo's LX2020 shows PKP2 protein +93%/+162% vs TN-401's +10% — if the field decides protein-expression magnitude predicts durable benefit, Tenaya is behind in ARVC, with Rocket also competing. Concentrated bet on being first and best in a three-way race.
• AAV cardiac safety is not settled. Reversible Grade 1–3 liver-enzyme elevations requiring immunosuppression are the norm; Rocket had a death (capillary leak) in a cardiac AAV program and an FDA hold. One serious AE in MyPEAK-1/RIDGE-1 (or a peer's trial) could halt the story. Patient 5 was already lost to follow-up.
• Most dangerous underestimated competitor: Lexeo in ARVC (ahead on biomarker, better capitalized relative to program breadth) and, in the broadest sense, the capital markets themselves — the entity that decides whether Tenaya survives.
• What must hold for $0.75 (let alone $3): (a) pivotal alignment on a fundable trial design in H2 2026; (b) a large raise or partnership on non-catastrophic terms; (c) no field-wide AAV safety shock; (d) TN-201 durability holds at 2 years. If growth/timelines disappoint by 20–30% (pivotal slips a year, raise happens at $0.40), the equity likely halves again despite intact science.
• The one scenario that permanently impairs it: a serious/fatal AAV-related SAE in either lead program → clinical hold → forced raise into a broken tape → dilution to a stub or restructuring. Plausibility: low-to-moderate but non-trivial, and it is the fat tail the $80M EV is (correctly) pricing.

Lens 14 · Management Questions (ordered by information value)

1. How, specifically, do you fund the TN-201 and TN-401 pivotal trials — dilutive equity, a development/commercial partnership, or non-dilutive (grants/royalty)? What is the trigger and the sequencing versus the H2-2026 alignment update? (The answer that most changes the thesis.)
2. What is your plan to regain Nasdaq minimum-bid compliance before July 27 — reverse split, transfer to the Capital Market, or an expected organic recovery — and what ratio are you contemplating?
3. At the current cash burn, what is your precise runway end-date including Alnylam reimbursements, and what is the minimum raise you'd accept versus terms you'd walk from?
4. On TN-401, how do you interpret your ~10% PKP2 protein increase against Lexeo's reported +93–162% — is functional arrhythmia benefit or protein-expression magnitude the better predictor of durable efficacy, and what does that imply for your pivotal endpoint?
5. What pivotal endpoint and trial size do you expect FDA/EMA to accept for TN-201 (surrogate like LVMI vs functional), and what would that trial cost and take?
6. Given Rocket's RP-A501 death and FDA hold, what is different about your AAV9 dose, immunosuppression regimen, and patient selection that limits capillary-leak/complement and hepatotoxicity risk?
7. Is a strategic partnership or outright sale of the platform (or TN-201) on the table, and how do you weigh that against dilutive going-it-alone?
8. What is the durability expectation for MyBP-C and PKP2 protein expression — does the biopsy trajectory support a truly one-time therapy over 5–10 years?
9. How do you prioritize capital across three clinical programs — would you pause TN-401 or TN-301 to concentrate cash on TN-201 if forced?
10. What is the realized value and probability profile of the Alnylam milestones, and what is the earliest development milestone that could pay cash?
11. What is current insider ownership and have insiders bought in the open market at these levels?
12. What are the manufacturing economics of the Union City plant at commercial scale, and is owning it still the right call versus a CDMO given the cash constraint?
13. What is your commercial/reimbursement thesis for pricing a one-time cardiac gene therapy, and what payer evidence are you building now?
14. Which single upcoming data point (2-yr Cohort 1 TN-201, TN-401 1-yr, or pivotal alignment) do you believe is most likely to re-rate the equity, and why hasn't good data moved the stock so far?
15. If the financing window stays shut through 2026, what is the contingency — restructuring, further layoffs, program prioritization, or an emergency partnership — and at what point do you decide?

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