REGENXBIO's RGX-121 BLA Accepted by FDA Under Accelerated Approval Pathway
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Feb 10 2026
0mins
Should l Buy RGNX?
Source: Benzinga
- FDA Approval Update: The FDA has accepted REGENXBIO's RGX-121 BLA under the accelerated approval pathway; however, the PDUFA goal date has been extended from November 9, 2025, to February 8, 2026, indicating the agency's cautious approach to expedited approvals.
- Reasons for CRL: The FDA issued a Complete Response Letter (CRL) for RGX-121, citing concerns regarding study eligibility criteria and the appropriateness of a surrogate endpoint, suggesting that new studies and long-term follow-ups may be necessary, which poses challenges in an ultra-rare disease population.
- Market Reaction: Despite regulatory hurdles, REGENXBIO's stock shows strong momentum in the market, with analysts maintaining a
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
7 Analyst Rating
7 Buy
0 Hold
0 Sell
Strong Buy
Current: 8.750
Low
19.00
Averages
29.71
High
45.00
Current: 8.750
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 to Host Conference Call for Q4 and Full Year Results
- Conference Call Announcement: REGENXBIO will host a conference call on March 5, 2026, at 8:00 a.m. ET to discuss its Q4 and full-year financial results for the year ended December 31, 2025, which is expected to attract significant attention from investors and analysts.
- Webcast Registration: Listeners can register for the webcast via the provided link, while analysts wishing to participate in the Q&A session must use the designated link, ensuring transparency and interactivity in the discussion.
- Replay Availability: A replay of the webcast will be available approximately two hours after the call concludes on the company's investor website, facilitating access to key information for investors who cannot participate live, thereby enhancing information accessibility.
- Company Background: Since its founding in 2009, REGENXBIO has been dedicated to improving lives through gene therapy, advancing a late-stage pipeline targeting rare and retinal diseases, showcasing its innovative potential and market impact in the biotechnology sector.
See More
REGENXBIO Faces Securities Litigation Investigation
- Legal Investigation Initiated: Faruq & Faruq, LLP is investigating potential securities litigation against REGENXBIO, concerning investor losses from February 9, 2022, to January 27, 2026, urging investors to contact them before the April 14, 2026 deadline to seek lead plaintiff status.
- FDA Clinical Hold: On January 28, 2026, REGENXBIO announced that the FDA placed a clinical hold on its gene therapies RGX-111 and RGX-121 due to a neoplasm case in a trial participant, causing the stock price to plummet by 17.9% to $11.01 per share, indicating investor concerns over the safety and efficacy of its products.
- Investor Rights Protection: Faruq & Faruq emphasizes that any investor wishing to participate in the lawsuit can apply to become a lead plaintiff through their chosen counsel, or opt out, ensuring their rights to potential recovery remain intact, reflecting transparency in legal proceedings.
- Information Gathering Appeal: The firm encourages anyone with information regarding REGENXBIO's conduct, including whistleblowers and former employees, to reach out, demonstrating a commitment to corporate governance and transparency aimed at protecting investors' legal rights.
See More
FDA Raises Concerns Over Invasive Gene Therapies
- Regulatory Pressure Intensifies: FDA Commissioner Marty Makary's concerns over invasive gene therapies led to a 31% drop in UniQure's AMT-130 stock, hitting a five-month low, indicating investor panic over regulatory prospects that could impact the company's funding and R&D efforts.
- Divergent Analyst Opinions: While RBC Capital Markets views Makary's comments as directed at UniQure, Leerink suggests they may refer to Regenxbio's RGX-121, reflecting market uncertainty regarding FDA regulatory policies that could erode investor confidence.
- Clinical Trial Challenges: Regenxbio's RGX-121 recently faced a Complete Response Letter from the FDA, citing uncertainties around study population definitions and surrogate endpoints, further heightening market concerns about its future development and competitive position.
- Opportunities in Personalized Therapies: Despite challenges, the FDA's recent draft guidance offers potential accelerated approvals for highly personalized gene therapies, indicating regulatory flexibility in treating ultra-rare conditions, which may present new growth opportunities for related companies.
See More
Class Action Lawsuits Filed Against PayPal and REGENXBIO
- PayPal Lawsuit Overview: A class action lawsuit against PayPal alleges that from February 25, 2025, to February 2, 2026, the company misled investors about its revenue outlook, resulting in a stock price drop of over 20% following disappointing earnings announced on February 3, 2026.
- CEO Transition Impact: The lawsuit highlights the transition of CEO James Alexander Chriss during the earnings announcement, exacerbating market concerns about the company's future and reflecting instability in management.
- REGENXBIO Lawsuit Background: The class action against REGENXBIO involves its gene therapy RGX-111, accusing the company of disseminating false information about trial success from February 9, 2022, to January 27, 2026, with the FDA placing a clinical hold on January 28, 2026, causing a 17.8% stock price drop in one day.
- Market Reaction Analysis: Both lawsuits indicate a decline in investor confidence regarding the companies' future prospects, with stock price volatility for PayPal and REGENXBIO reflecting heightened market scrutiny on corporate governance and operational transparency, potentially affecting their long-term investment appeal.
See More
Reminder of REGENXBIO Securities Class Action
- Class Action Notice: Rosen Law Firm reminds investors who purchased REGENXBIO (NASDAQ: RGNX) securities between February 9, 2022, and January 27, 2026, to apply as lead plaintiffs by April 14, 2026, to participate in the class action and seek compensation.
- Fee Arrangement: Investors joining the class action will incur no out-of-pocket costs, as the law firm operates on a contingency fee basis, ensuring legal support without financial burden for the investors.
- Case Background: The lawsuit alleges that defendants provided false and misleading information regarding the development of REGENXBIO's RGX-111 gene therapy, resulting in investor losses when the true details emerged, highlighting the importance of protecting investor rights.
- Law Firm Credentials: Rosen Law Firm specializes in securities class actions and recovered over $438 million for investors in 2019 alone, being ranked first in 2017 for the number of securities class action settlements, demonstrating its expertise and success in this field.
See More
FDA Tightens Stance on Rare Disease Drug Approvals
- FDA Policy Tightening: In an interview, FDA Commissioner Makary defended the agency's stricter stance on rare disease drug approvals, mentioning a product that showed no benefit in a randomized trial and had associated morbidity, indicating a more cautious approach that could slow future drug approvals.
- uniQure Stock Plunge: Following Makary's comments, uniQure's shares plummeted 32% on Thursday, reflecting market concerns over the approval prospects of its Huntington's disease gene therapy AMT-130, especially after the FDA deemed early trial data insufficient to support an application.
- Market Sentiment Shift: Despite the stock drop, retail sentiment around QURE on Stocktwits shifted from 'bearish' to 'bullish' within 24 hours, indicating investor optimism about the company's plans to communicate with the FDA regarding a path forward for approval.
- Competitive Drug Analysis: Analysts noted that Makary's comments could refer to either uniQure's AMT-130 or Regenxbio's RGX-121, the latter of which was not approved by the FDA due to insufficient evidence of effectiveness, highlighting the agency's rigorous scrutiny that may impact the entire industry's R&D direction.
See More
REGENXBIO to Host Conference Call for Q4 and Full Year Results
- Conference Call Announcement: REGENXBIO will host a conference call on March 5, 2026, at 8:00 a.m. ET to discuss its Q4 and full-year financial results for the year ended December 31, 2025, which is expected to attract significant attention from investors and analysts.
- Webcast Registration: Listeners can register for the webcast via the provided link, while analysts wishing to participate in the Q&A session must use the designated link, ensuring transparency and interactivity in the discussion.
- Replay Availability: A replay of the webcast will be available approximately two hours after the call concludes on the company's investor website, facilitating access to key information for investors who cannot participate live, thereby enhancing information accessibility.
- Company Background: Since its founding in 2009, REGENXBIO has been dedicated to improving lives through gene therapy, advancing a late-stage pipeline targeting rare and retinal diseases, showcasing its innovative potential and market impact in the biotechnology sector.
See More
REGENXBIO Faces Securities Litigation Investigation
- Legal Investigation Initiated: Faruq & Faruq, LLP is investigating potential securities litigation against REGENXBIO, concerning investor losses from February 9, 2022, to January 27, 2026, urging investors to contact them before the April 14, 2026 deadline to seek lead plaintiff status.
- FDA Clinical Hold: On January 28, 2026, REGENXBIO announced that the FDA placed a clinical hold on its gene therapies RGX-111 and RGX-121 due to a neoplasm case in a trial participant, causing the stock price to plummet by 17.9% to $11.01 per share, indicating investor concerns over the safety and efficacy of its products.
- Investor Rights Protection: Faruq & Faruq emphasizes that any investor wishing to participate in the lawsuit can apply to become a lead plaintiff through their chosen counsel, or opt out, ensuring their rights to potential recovery remain intact, reflecting transparency in legal proceedings.
- Information Gathering Appeal: The firm encourages anyone with information regarding REGENXBIO's conduct, including whistleblowers and former employees, to reach out, demonstrating a commitment to corporate governance and transparency aimed at protecting investors' legal rights.
See More
FDA Raises Concerns Over Invasive Gene Therapies
- Regulatory Pressure Intensifies: FDA Commissioner Marty Makary's concerns over invasive gene therapies led to a 31% drop in UniQure's AMT-130 stock, hitting a five-month low, indicating investor panic over regulatory prospects that could impact the company's funding and R&D efforts.
- Divergent Analyst Opinions: While RBC Capital Markets views Makary's comments as directed at UniQure, Leerink suggests they may refer to Regenxbio's RGX-121, reflecting market uncertainty regarding FDA regulatory policies that could erode investor confidence.
- Clinical Trial Challenges: Regenxbio's RGX-121 recently faced a Complete Response Letter from the FDA, citing uncertainties around study population definitions and surrogate endpoints, further heightening market concerns about its future development and competitive position.
- Opportunities in Personalized Therapies: Despite challenges, the FDA's recent draft guidance offers potential accelerated approvals for highly personalized gene therapies, indicating regulatory flexibility in treating ultra-rare conditions, which may present new growth opportunities for related companies.
See More
Class Action Lawsuits Filed Against PayPal and REGENXBIO
- PayPal Lawsuit Overview: A class action lawsuit against PayPal alleges that from February 25, 2025, to February 2, 2026, the company misled investors about its revenue outlook, resulting in a stock price drop of over 20% following disappointing earnings announced on February 3, 2026.
- CEO Transition Impact: The lawsuit highlights the transition of CEO James Alexander Chriss during the earnings announcement, exacerbating market concerns about the company's future and reflecting instability in management.
- REGENXBIO Lawsuit Background: The class action against REGENXBIO involves its gene therapy RGX-111, accusing the company of disseminating false information about trial success from February 9, 2022, to January 27, 2026, with the FDA placing a clinical hold on January 28, 2026, causing a 17.8% stock price drop in one day.
- Market Reaction Analysis: Both lawsuits indicate a decline in investor confidence regarding the companies' future prospects, with stock price volatility for PayPal and REGENXBIO reflecting heightened market scrutiny on corporate governance and operational transparency, potentially affecting their long-term investment appeal.
See More
Reminder of REGENXBIO Securities Class Action
- Class Action Notice: Rosen Law Firm reminds investors who purchased REGENXBIO (NASDAQ: RGNX) securities between February 9, 2022, and January 27, 2026, to apply as lead plaintiffs by April 14, 2026, to participate in the class action and seek compensation.
- Fee Arrangement: Investors joining the class action will incur no out-of-pocket costs, as the law firm operates on a contingency fee basis, ensuring legal support without financial burden for the investors.
- Case Background: The lawsuit alleges that defendants provided false and misleading information regarding the development of REGENXBIO's RGX-111 gene therapy, resulting in investor losses when the true details emerged, highlighting the importance of protecting investor rights.
- Law Firm Credentials: Rosen Law Firm specializes in securities class actions and recovered over $438 million for investors in 2019 alone, being ranked first in 2017 for the number of securities class action settlements, demonstrating its expertise and success in this field.
See More
FDA Tightens Stance on Rare Disease Drug Approvals
- FDA Policy Tightening: In an interview, FDA Commissioner Makary defended the agency's stricter stance on rare disease drug approvals, mentioning a product that showed no benefit in a randomized trial and had associated morbidity, indicating a more cautious approach that could slow future drug approvals.
- uniQure Stock Plunge: Following Makary's comments, uniQure's shares plummeted 32% on Thursday, reflecting market concerns over the approval prospects of its Huntington's disease gene therapy AMT-130, especially after the FDA deemed early trial data insufficient to support an application.
- Market Sentiment Shift: Despite the stock drop, retail sentiment around QURE on Stocktwits shifted from 'bearish' to 'bullish' within 24 hours, indicating investor optimism about the company's plans to communicate with the FDA regarding a path forward for approval.
- Competitive Drug Analysis: Analysts noted that Makary's comments could refer to either uniQure's AMT-130 or Regenxbio's RGX-121, the latter of which was not approved by the FDA due to insufficient evidence of effectiveness, highlighting the agency's rigorous scrutiny that may impact the entire industry's R&D direction.
See More
