Regenxbio's RGX-121 Faces FDA Approval Setback
Written by Emily J. Thompson, Senior Investment Analyst
Updated: 2h ago
0mins
Should l Buy RGNX?
Source: stocktwits
- FDA Rejection: The FDA issued a Complete Response Letter (CRL) for Regenxbio's RGX-121 gene therapy, citing insufficient evidence of effectiveness, which led to a nearly 16% drop in the company's stock price after hours, significantly impacting its future market performance.
- Price Target Adjustment: Clear Street lowered its price target for Regenxbio from $50 to $45, yet maintained a 'Buy' rating, indicating confidence in the company's long-term potential and suggesting over a 300% upside from the current stock price.
- BLA Resubmission Plans: Regenxbio plans to hold a Type A meeting with the FDA to discuss the CRL and resubmission strategy for its Biologics License Application (BLA), aiming to provide additional evidence from global MPS II experts to support the therapy's effectiveness and expedite the approval process.
- Market Sentiment Bearish: On Stocktwits, retail sentiment for RGNX is described as 'extremely bearish', with users expressing concerns about the company's need for further financing and the FDA's additional requirements, reflecting a lack of confidence in the product's future prospects.
Trade with 70% Backtested Accuracy
Stop guessing "Should I Buy RGNX?" and start using high-conviction signals backed by rigorous historical data.
Sign up today to access powerful investing tools and make smarter, data-driven decisions.
Analyst Views on RGNX
Wall Street analysts forecast RGNX stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for RGNX is 29.71 USD with a low forecast of 19.00 USD and a high forecast of 45.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
7 Analyst Rating
7 Buy
0 Hold
0 Sell
Strong Buy
Current: 10.330
Low
19.00
Averages
29.71
High
45.00
Current: 10.330
Low
19.00
Averages
29.71
High
45.00
About RGNX
REGENXBIO Inc. is a clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. The Company has developed a pipeline of gene therapy programs using its proprietary adeno-associated virus (AAV) gene therapy delivery platform (NAV Technology Platform) to address genetic diseases. It is focused on its internal development pipeline in three areas: retinal, neuromuscular, and neurodegenerative diseases. Its investigational AAV therapeutics include ABBV-RGX-314, RGX-202, RGX-121, and RGX-111. It is developing ABBV-RGX-314 in collaboration with AbbVie to treat large patient populations impacted by wet age-related macular degeneration, diabetic retinopathy (DR) and other chronic retinal diseases characterized by loss of vision. It is developing RGX-202 to treat Duchenne muscular dystrophy (Duchenne). The Company is developing RGX-121 to treat Mucopolysaccharidosis type II (MPS II), a progressive, neurodegenerative lysosomal storage disorder.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Regenxbio's RGX-121 Faces FDA Approval Setback
- FDA Rejection: The FDA issued a Complete Response Letter (CRL) for Regenxbio's RGX-121 gene therapy, citing insufficient evidence of effectiveness, which led to a nearly 16% drop in the company's stock price after hours, significantly impacting its future market performance.
- Price Target Adjustment: Clear Street lowered its price target for Regenxbio from $50 to $45, yet maintained a 'Buy' rating, indicating confidence in the company's long-term potential and suggesting over a 300% upside from the current stock price.
- BLA Resubmission Plans: Regenxbio plans to hold a Type A meeting with the FDA to discuss the CRL and resubmission strategy for its Biologics License Application (BLA), aiming to provide additional evidence from global MPS II experts to support the therapy's effectiveness and expedite the approval process.
- Market Sentiment Bearish: On Stocktwits, retail sentiment for RGNX is described as 'extremely bearish', with users expressing concerns about the company's need for further financing and the FDA's additional requirements, reflecting a lack of confidence in the product's future prospects.
See More
REGENXBIO's Gene Therapy Denied by FDA
- FDA Denial Reasons: The FDA cited uncertainty in eligibility criteria for distinguishing between neuronopathic and attenuated disease populations as a key reason for denying REGENXBIO's gene therapy application, raising concerns about the trial's validity.
- Stock Price Plunge: Following the FDA's rejection of its gene therapy for Hunter syndrome, REGENXBIO's shares plummeted approximately 19% in after-hours trading, indicating significant market pessimism regarding the company's future prospects.
- Clinical Hold Imposed: Last month, the FDA placed a clinical hold on two of REGENXBIO's experimental gene therapy programs, exacerbating investor concerns about the company's research progress, particularly in treating rare childhood diseases.
- Path Forward Plans: REGENXBIO intends to meet with the FDA to address the Complete Response Letter feedback, gather expert input, and better define the eligible patient population, aiming to resubmit its application as swiftly as possible to advance its treatment strategy.
See More
Regenxbio Faces FDA Clinical Hold Investigation
- FDA Clinical Hold: Regenxbio's investigational gene therapy RGX-111 has been placed on clinical hold by the FDA due to a neoplasm case, significantly hindering the company's progress in treating MPS I and potentially stalling future research efforts.
- Stock Price Plunge: Following the FDA's announcement, Regenxbio's stock price fell by $2.40, or 17.9%, closing at $11.01 per share, indicating market concerns regarding the company's future prospects.
- Legal Investigation Initiated: Pomerantz LLP is investigating whether Regenxbio and its executives have engaged in securities fraud or other unlawful business practices, which could expose the company to additional legal risks and financial liabilities.
- Investor Advisory: Investors are advised to contact Pomerantz LLP for information on joining the class action, highlighting the market's heightened scrutiny of the company's governance and compliance practices.
See More
Regenxbio Faces Investigation Over FDA Clinical Holds
- FDA Clinical Hold: On January 28, 2026, Regenxbio disclosed that the FDA placed clinical holds on its RGX-111 and RGX-121 programs due to a tumor found in a trial participant, resulting in a 30-35% drop in share price, severely impacting investor confidence.
- Disclosure Gaps: During the Q3 2025 earnings call, CEO Curran Simpson highlighted favorable FDA inspection results but failed to mention safety concerns regarding the RGX-111 program, creating an asymmetric information presentation that may violate SEC disclosure rules.
- Investor Rights at Risk: The lack of updates on the RGX-111 program raises concerns among investors about the company's future, potentially leading more shareholders to seek legal recourse to protect their interests, thereby increasing the company's legal risks.
- Legal Investigation Launched: Levi & Korsinsky is investigating Regenxbio for potential gaps between the information provided to investors and actual safety developments, which could impact the company's reputation and future financing capabilities.
See More
Regenxbio Faces FDA Clinical Hold Investigation
- FDA Clinical Hold: On January 28, 2026, Regenxbio disclosed that the FDA placed clinical holds on its RGX-111 and RGX-121 programs due to a tumor found in a trial participant, resulting in a 30-35% drop in share price, severely impacting investor confidence.
- Disclosure Gaps: During the Q3 2025 earnings call, CEO Curran Simpson highlighted favorable FDA inspection results but failed to mention safety concerns regarding the RGX-111 program, creating an asymmetric presentation that may violate SEC disclosure rules.
- Investor Rights Affected: The lack of updates on the RGX-111 program raises doubts among investors about the company's future, potentially leading more shareholders to seek legal recourse, thereby increasing the company's legal risks.
- Legal Investigation Initiated: Levi & Korsinsky is investigating Regenxbio for potential significant omissions in the information provided to investors, which could affect the company's reputation in the securities market and its future financing capabilities.
See More
REGENXBIO Faces FDA Clinical Hold on Gene Therapy RGX-111
- FDA Clinical Hold: REGENXBIO's gene therapy RGX-111 for Hurler syndrome faces a clinical hold from the FDA due to a tumor case in a trial participant, causing shares to plummet approximately 30% in premarket trading, highlighting market sensitivity to FDA unpredictability.
- Analyst Perspectives: Clear Street analyst Bill Maughan reiterated a ‘Buy’ rating with a $50 price target, noting that the causality between RGX-111 and the tumor remains unestablished, and the patient was asymptomatic, suggesting the market's reaction may be an overreaction.
- Market Reaction: Despite the stock's decline post-announcement, Leerink views the selloff as excessive, believing that the financial impact of Hunter/Hurler syndrome on the company's fundamental value is modest, thus presenting a buying opportunity while maintaining an ‘Outperform’ rating.
- Future Outlook: Stifel also reaffirmed a ‘Buy’ rating with a $45 price target, expecting a delay in FDA approval for RGX-121, yet maintains that the risk-benefit profile for other gene therapies remains favorable.
See More
Regenxbio's RGX-121 Faces FDA Approval Setback
- FDA Rejection: The FDA issued a Complete Response Letter (CRL) for Regenxbio's RGX-121 gene therapy, citing insufficient evidence of effectiveness, which led to a nearly 16% drop in the company's stock price after hours, significantly impacting its future market performance.
- Price Target Adjustment: Clear Street lowered its price target for Regenxbio from $50 to $45, yet maintained a 'Buy' rating, indicating confidence in the company's long-term potential and suggesting over a 300% upside from the current stock price.
- BLA Resubmission Plans: Regenxbio plans to hold a Type A meeting with the FDA to discuss the CRL and resubmission strategy for its Biologics License Application (BLA), aiming to provide additional evidence from global MPS II experts to support the therapy's effectiveness and expedite the approval process.
- Market Sentiment Bearish: On Stocktwits, retail sentiment for RGNX is described as 'extremely bearish', with users expressing concerns about the company's need for further financing and the FDA's additional requirements, reflecting a lack of confidence in the product's future prospects.
See More
REGENXBIO's Gene Therapy Denied by FDA
- FDA Denial Reasons: The FDA cited uncertainty in eligibility criteria for distinguishing between neuronopathic and attenuated disease populations as a key reason for denying REGENXBIO's gene therapy application, raising concerns about the trial's validity.
- Stock Price Plunge: Following the FDA's rejection of its gene therapy for Hunter syndrome, REGENXBIO's shares plummeted approximately 19% in after-hours trading, indicating significant market pessimism regarding the company's future prospects.
- Clinical Hold Imposed: Last month, the FDA placed a clinical hold on two of REGENXBIO's experimental gene therapy programs, exacerbating investor concerns about the company's research progress, particularly in treating rare childhood diseases.
- Path Forward Plans: REGENXBIO intends to meet with the FDA to address the Complete Response Letter feedback, gather expert input, and better define the eligible patient population, aiming to resubmit its application as swiftly as possible to advance its treatment strategy.
See More
Regenxbio Faces FDA Clinical Hold Investigation
- FDA Clinical Hold: Regenxbio's investigational gene therapy RGX-111 has been placed on clinical hold by the FDA due to a neoplasm case, significantly hindering the company's progress in treating MPS I and potentially stalling future research efforts.
- Stock Price Plunge: Following the FDA's announcement, Regenxbio's stock price fell by $2.40, or 17.9%, closing at $11.01 per share, indicating market concerns regarding the company's future prospects.
- Legal Investigation Initiated: Pomerantz LLP is investigating whether Regenxbio and its executives have engaged in securities fraud or other unlawful business practices, which could expose the company to additional legal risks and financial liabilities.
- Investor Advisory: Investors are advised to contact Pomerantz LLP for information on joining the class action, highlighting the market's heightened scrutiny of the company's governance and compliance practices.
See More
Regenxbio Faces Investigation Over FDA Clinical Holds
- FDA Clinical Hold: On January 28, 2026, Regenxbio disclosed that the FDA placed clinical holds on its RGX-111 and RGX-121 programs due to a tumor found in a trial participant, resulting in a 30-35% drop in share price, severely impacting investor confidence.
- Disclosure Gaps: During the Q3 2025 earnings call, CEO Curran Simpson highlighted favorable FDA inspection results but failed to mention safety concerns regarding the RGX-111 program, creating an asymmetric information presentation that may violate SEC disclosure rules.
- Investor Rights at Risk: The lack of updates on the RGX-111 program raises concerns among investors about the company's future, potentially leading more shareholders to seek legal recourse to protect their interests, thereby increasing the company's legal risks.
- Legal Investigation Launched: Levi & Korsinsky is investigating Regenxbio for potential gaps between the information provided to investors and actual safety developments, which could impact the company's reputation and future financing capabilities.
See More
Regenxbio Faces FDA Clinical Hold Investigation
- FDA Clinical Hold: On January 28, 2026, Regenxbio disclosed that the FDA placed clinical holds on its RGX-111 and RGX-121 programs due to a tumor found in a trial participant, resulting in a 30-35% drop in share price, severely impacting investor confidence.
- Disclosure Gaps: During the Q3 2025 earnings call, CEO Curran Simpson highlighted favorable FDA inspection results but failed to mention safety concerns regarding the RGX-111 program, creating an asymmetric presentation that may violate SEC disclosure rules.
- Investor Rights Affected: The lack of updates on the RGX-111 program raises doubts among investors about the company's future, potentially leading more shareholders to seek legal recourse, thereby increasing the company's legal risks.
- Legal Investigation Initiated: Levi & Korsinsky is investigating Regenxbio for potential significant omissions in the information provided to investors, which could affect the company's reputation in the securities market and its future financing capabilities.
See More
REGENXBIO Faces FDA Clinical Hold on Gene Therapy RGX-111
- FDA Clinical Hold: REGENXBIO's gene therapy RGX-111 for Hurler syndrome faces a clinical hold from the FDA due to a tumor case in a trial participant, causing shares to plummet approximately 30% in premarket trading, highlighting market sensitivity to FDA unpredictability.
- Analyst Perspectives: Clear Street analyst Bill Maughan reiterated a ‘Buy’ rating with a $50 price target, noting that the causality between RGX-111 and the tumor remains unestablished, and the patient was asymptomatic, suggesting the market's reaction may be an overreaction.
- Market Reaction: Despite the stock's decline post-announcement, Leerink views the selloff as excessive, believing that the financial impact of Hunter/Hurler syndrome on the company's fundamental value is modest, thus presenting a buying opportunity while maintaining an ‘Outperform’ rating.
- Future Outlook: Stifel also reaffirmed a ‘Buy’ rating with a $45 price target, expecting a delay in FDA approval for RGX-121, yet maintains that the risk-benefit profile for other gene therapies remains favorable.
See More
