Rocket Pharmaceuticals Inc.

RCKT

Held by 2 specialist biotech funds

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Fund Holdings

Driehaus Capital RTW Investments

AI Signal

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Catalysts

No catalysts tracked.

Recent News & Research

Curated

Rocket Pharma Gets FDA Approval for First Gene Therapy for LAD-I

Rocket Pharmaceuticals received accelerated FDA approval for Kresladi (marnetegragene autotemcel), the first gene therapy option for severe leukocyte adhesion deficiency-I. The approval came ahead of the March 28 PDUFA date, positioning Rocket as a leader in ultra-rare immune disease treatment.

Mar 29, 2026

Curated

FDA grants accelerated approval to Rocket Pharmaceuticals Kresladi for severe LAD-I

Position Details

Driehaus CapitalNEWFeb 17, 2026

Shares: 393,430Value: $27.4M

**Signal Note: Driehaus Capital initiates $27.4M position in Rocket Pharmaceuticals** Driehaus's entry suggests confidence in Rocket's gene therapy pipeline, likely driven by near-term catalysts in its lead programs: RP-L102 for Danon disease and RP-L201 for aromatic L-amino acid decarboxylase (AADC) deficiency, both addressing ultra-rare cardiac and neurological indications with limited alternatives. The $27.4M stake positions the firm ahead of potential clinical data readouts or regulatory milestones expected through 2024-2025, where gene therapies in these segments command significant risk-adjusted valuations given high unmet need and commercial exclusivity potential.

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Also Held by These Funds

NBIX2 funds CBIO2 funds PODD2 funds TARS2 funds BBNX2 funds LRMR2 funds SYRE2 funds ARWR

Research Links

Yahoo Finance →SEC EDGAR →ClinicalTrials.gov →

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**Signal Note: RTW Investments initiates $62.1M stake in RCKT** RTW's entry into Rocket Pharmaceuticals signals confidence in the company's late-stage pipeline, likely driven by upcoming clinical readouts or regulatory catalysts in their lentiviral gene therapy programs (notably RP-L102 for frangible bone disease and RP-M01 for mucopolysaccharidosis). The $62.1M position (18M shares) represents a meaningful conviction bet from a data-focused investor, typically indicating visible near-term milestones or competitive moat thesis rather than speculative upside.