Regenxbio’s Two Lead Gene Therapies Back Mizuho’s Nearly 200% Upside Call

Mizuho initiated coverage of Regenxbio with an ‘Outperform’ rating and a $22 price target.

• The brokerage flagged RGX-202 for DMD and Sura-vec for wet AMD as two major potential catalysts for the stock.
• Regenxbio’s Phase 3 Affinity Duchenne study met its primary endpoint earlier this year.
• Regenxbio expects topline Phase 3 data from the Atmosphere and Ascent wet AMD studies in Q4 of 2026.

Regenxbio (RGNX) was in the spotlight on Monday, with Mizuho highlighting two late-stage gene therapy assets as key drivers of 196% upside potential from current levels.

The brokerage initiated coverage with an ‘Outperform’ rating and a $22 price target, according to The Fly. This is slightly lower than the 12-month consensus price target of $23.73, according to Koyfin. Nine of the 11 analysts covering the stock have rated it a ‘Buy,’ while two have it rated ‘Hold.’

RGNX shares jumped 6.5% higher in pre-market trading. Last week, the stock clocked its first weekly gain since mid-August.

RGX-202 For DMD Is A Major Potential Catalyst

RegenxBio’s RGX-202 is designed to deliver a novel Microdystrophin gene to patients with Duchenne muscular dystrophy (DMD), a severe muscle disease caused by mutations in the dystrophin gene. Regenxbio’s Phase 3 Affinity Duchenne study met its primary endpoint earlier this year, with 93% of patients achieving Microdystrophin expression above 10%.

The company had previously mentioned plans to initiate a Biologics License Application (BLA) under the accelerated approval pathway, with potential U.S. Food and Drug Administration (FDA) approval in the second half of 2027.

Sura-Vec Adds A Second Major Catalyst

Mizuho also flagged RegenxBio’s Sura-Vec, which is being developed in partnership with AbbVie, as a one-time gene therapy for wet age-related macular degeneration and diabetic retinopathy.

Regenxbio expects topline Phase 3 data from the Atmosphere and Ascent wet AMD studies in the fourth quarter (Q4) of 2026. The company has also started a Phase 2b/3 diabetic retinopathy study.

RGX-121 Remains A Pipeline Risk 

The company’s broader pipeline also includes RGX-121 for Hunter syndrome, although the program was placed on clinical hold in August and the company said it does not expect to resubmit its BLA in the near term.

Cash Position Gives RGNX More Runway 

As of June 30, Regenxbio had $105.5 million in cash, cash equivalents and marketable securities. Its financial position strengthened further in July after the company received a $100 million milestone payment from AbbVie and raised about $108 million through an equity offering, taking its pro forma liquidity to more than $300 million. This extends its expected cash runway into Q4 2027.

Retail’s Take On RGNX

Retail sentiment for RGNX on Stocktwits remained in the ‘bullish’ zone over the past 24 hours.

One user highlighted job listings by Regenxbio for sales positions related to its DMD drug.

View this Stocktwits post

However, the stock has faced selling pressure this year, down more than 45%.

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