Full-Time
Senior Medical Science Liaison
DMD/Neuromuscular
REGENXBIO
201-500 employees
Develops AAV gene delivery therapies
Compensation Overview
$170k - $230k/yr
Remote in USA
Remote
Master's, PharmD, PhD, MD
 Biology & Biotech (1) |
|---|
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- Master’s degree (MPH or equivalent) required
- Minimum of 3 years of rare disease clinical or therapeutic experience in neuromuscular diseases, and/ or gene therapy with primary emphasis on field based scientific exchange and/or clinical trial support
- Superior communication, relationship building and interpersonal skills
- Ability to assimilate new clinical data quickly
- Proactive problem-solving skills
- Project management and medical writing skills
- Flexibility, resilience, and creativity/ innovation in their work
- Ability to organize and lead high level projects
- Ability to proactively predict issues and solve problems
- Ability to work collaboratively with cross-functional teams
- Support clinical trial recruitment and site outreach; communicate coordinated feedback to the Clinical Operations, Clinical Development, and Duchenne Core teams
- Serve as the field-based Medical Affairs representative to establish and cultivate scientific relationships with KOLs in the Duchenne community
- Develop a strong understanding of clinical practice as it relates to Duchenne
- Participate in refining KOL mapping; develop and execute on a customer-centric tactical engagement plan to support Medical Affairs strategies
- Gather and share scientific insights via scientific exchange to inform executable and innovative Medical Affairs strategies
- Serve as a resource for addressing unsolicited medical questions about REGENXBIO’s trials and investigational products
- Provide clinical expertise in the development, management, and maintenance of clinical and scientific communications, including educational materials, meeting presentations and FAQs
- Attend medical conferences as MA representative: assist with congress strategy / planning, medical booth staffing, competitive intelligence / insights gathering, and generating meeting summaries
- Support Patient Advocacy conferences and outreach as requested
- As a field-based position, you must be able to travel 50-70% of the time
- Doctorate level (MD, PharmD, PhD) preferred
REGENXBIO focuses on gene therapy using its NAV Technology Platform, an adeno-associated virus (AAV) gene delivery system with exclusive rights to more than 100 AAV vectors. The company generates revenue by licensing its platform, collecting royalties, and engaging in collaborations, while also developing its own one-time gene therapies for retinal, rare, and neurodegenerative diseases. Key products in later-stage trials include ABBV-RGX-314 for wet AMD and diabetic retinopathy, RGX-202 for Duchenne muscular dystrophy, and RGX-121 for Hunter syndrome, with a goal to advance five new therapies to pivotal-stage or commercial status by 2025 and FDA review for RGX-121 targeted in early 2026. REGENXBIO differentiates itself by owning a broad AAV vector library and offering end-to-end capabilities—from research to manufacturing—alongside a diversified revenue model that includes royalties, collaborations, and milestones.
Company Size
201-500
Company Stage
IPO
Headquarters
Rockville, Maryland
Founded
2009
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Simplify's Take
What believers are saying
- RGX-202 met its primary endpoint; REGENXBIO targets BLA submission in Q3 2026.
- FDA backed RGX-121 accelerated-approval resubmission; management plans filing in Q3 2026.
- Over $200 million July financing lifted cash runway into Q4 2027 ahead of Q4 2026 data.
What critics are saying
- FDA clinical hold on RGX-111 after a CNS tumor threatens RGX-121 and platform credibility.
- Securities class action over RGX-111 safety disclosures creates settlement costs and distraction through 2026.
- Zolgensma royalty revenue fell after January 2026 patent expiry, exposing dependence on one-time catalysts.
What makes REGENXBIO unique
- NAV platform controls over 100 AAV vectors, including AAV7, AAV8, and AAV9.
- AbbVie and Nippon Shinyaku validate REGENXBIO’s platform with partnered ocular and rare-disease programs.
- The company owns end-to-end gene-therapy capabilities across discovery, manufacturing, and late-stage development.
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Benefits
401(k) Retirement Plan
401(k) Company Match
Stock Options
Performance Bonus
Professional Development Budget
Growth & Insights and Company News
6 month growth
↑ 1%1 year growth
↑ 2%2 year growth
↑ 0%REGENXBIO (NASDAQ:RGNX) shares down 6.1% - Here's why. August 7, 2026 Key points. * REGENXBIO shares fell 6.1% to about $10.59 despite reporting second-quarter adjusted EPS of $0.43 and revenue of $108.02 million, both well above analyst expectations. * The company raised more than $200 million in July, extending its cash runway into late 2027, while key regulatory and clinical milestones - including RGX-202, RGX-121 and RGX-314 updates - are expected in 2026-2027. * Investor sentiment remains mixed: analysts' consensus is "Moderate Buy" with a $23.82 price target, but REGENXBIO remains loss-making, expects a full-year loss, and its valuation depends heavily on successful clinical and regulatory outcomes. * MarketBeat previews the top five stocks to own by September 1st. REGENXBIO Inc. (NASDAQ:RGNX - Get Free Report) was down 6.1% on Friday. The stock traded as low as $10.62 and last traded at $10.5940. 194,626 shares traded hands during mid-day trading, a decline of 87% from the average daily volume of 1,452,026 shares. The stock had previously closed at $11.28. Key stories impacting REGENXBIO. Here are the key news stories impacting REGENXBIO this week: * Positive Sentiment: Q2 results exceeded expectations. REGENXBIO reported adjusted earnings of $0.43 per share, well above analyst estimates ranging from $0.08 to $0.12, while revenue of $108.02 million surpassed the $97.63 million consensus estimate. The result compared with a $1.38-per-share loss in the year-ago quarter. REGENXBIO Beats Q2 Earnings and Revenue Estimates * Positive Sentiment: Cash runway and regulatory milestones improved visibility. The company said more than $200 million of new capital raised in July extended its cash runway into the fourth quarter of 2027. The RGX-202 biologics license application for Duchenne muscular dystrophy remains on track to begin in the third quarter of 2026, with potential accelerated approval in the second half of 2027. REGENXBIO Q2 2026 Financial Results * Positive Sentiment: Multiple clinical catalysts remain ahead. Topline data from the ATMOSPHERE and ASCENT studies of ABBV-RGX-314 for wet age-related macular degeneration are expected in the fourth quarter of 2026. REGENXBIO also reaffirmed a third-quarter 2026 resubmission plan for RGX-121 in Hunter syndrome following an FDA Type A meeting. REGENXBIO Cash Runway and RGX-202 Update * Neutral Sentiment: Barclays maintained an "Equal Weight" rating but raised or reaffirmed a $12 price target, implying roughly 15% upside from the cited share price. The target suggests measured optimism rather than a strongly bullish view. Barclays Rating Update * Negative Sentiment: REGENXBIO remains financially loss-making. Despite the quarterly profit, the company reported a deeply negative net margin, and analysts still expect a full-year loss. The stock therefore remains highly dependent on successful clinical results, regulatory filings and future commercialization. Analyst Ratings changes. A number of analysts have commented on the company. Morgan Stanley reissued an "overweight" rating on shares of REGENXBIO in a report on Friday. Robert W. Baird raised their target price on REGENXBIO from $27.00 to $32.00 and gave the company an "outperform" rating in a report on Monday, June 22nd. Zacks Research cut REGENXBIO from a "hold" rating to a "strong sell" rating in a research report on Tuesday, July 14th. Weiss Ratings reiterated a "sell (e+)" rating on shares of REGENXBIO in a report on Monday, June 8th. Finally, Bank of America lowered their price target on REGENXBIO from $25.00 to $14.00 and set a "buy" rating for the company in a research report on Friday, May 15th. Nine research analysts have rated the stock with a Buy rating, two have given a Hold rating and two have given a Sell rating to the company's stock. According to data from MarketBeat, REGENXBIO currently has a consensus rating of "Moderate Buy" and an average price target of $23.82. Discover more Stock Average Calculator REGENXBIO price performance. The firm's fifty day moving average is $9.94 and its two-hundred day moving average is $9.42. The firm has a market capitalization of $535.61 million, a PE ratio of -1.86 and a beta of 0.94. REGENXBIO (NASDAQ:RGNX - Get Free Report) last posted its quarterly earnings data on Thursday, August 6th. The biotechnology company reported $0.43 EPS for the quarter, beating the consensus estimate of $0.08 by $0.35. REGENXBIO had a negative net margin of 330.23% and a negative return on equity of 232.50%. The firm had revenue of $108.02 million during the quarter, compared to analyst estimates of $97.63 million. As a group, equities research analysts predict that REGENXBIO Inc. will post -3.69 EPS for the current fiscal year. Insider activity. In related news, insider Steve Pakola sold 36,725 shares of the stock in a transaction that occurred on Wednesday, July 1st. The stock was sold at an average price of $12.86, for a total transaction of $472,283.50. Following the completion of the sale, the insider directly owned 213,009 shares of the company's stock, valued at approximately $2,739,295.74. This trade represents a 14.71% decrease in their ownership of the stock. The sale was disclosed in a legal filing with the SEC, which can be accessed through this link. Insiders sold 73,803 shares of company stock valued at $886,937 over the last three months. Corporate insiders own 14.22% of the company's stock. Institutional inflows and outflows. Several hedge funds and other institutional investors have recently bought and sold shares of the business. Versant Capital Management Inc increased its position in shares of REGENXBIO by 60.4% during the 2nd quarter. Versant Capital Management Inc now owns 2,741 shares of the biotechnology company's stock valued at $33,000 after purchasing an additional 1,032 shares during the last quarter. Aquatic Capital Management LLC purchased a new position in shares of REGENXBIO in the 3rd quarter worth about $37,000. Captrust Financial Advisors purchased a new position in shares of REGENXBIO in the 2nd quarter worth about $85,000. Entropy Technologies LP bought a new position in REGENXBIO during the first quarter valued at about $87,000. Finally, Prudential Financial Inc. bought a new position in REGENXBIO during the second quarter valued at about $87,000. Hedge funds and other institutional investors own 88.08% of the company's stock. REGENXBIO company profile. REGENXBIO Inc is a clinical-stage biotechnology company specializing in the development of gene therapies using its proprietary NAV(R) AAV (adeno-associated virus) platform. The company engineers next-generation AAV vectors designed to deliver functional genes to targeted cells, aiming to address a range of rare genetic diseases and ocular, metabolic and neurologic disorders. REGENXBIO's pipeline features several product candidates in various stages of preclinical and clinical development, including RGX-314 for wet age-related macular degeneration, RGX-121 for mucopolysaccharidosis II (Hunter syndrome) and RGX-121 for other rare lysosomal storage diseases. In addition to its internally funded programs, REGENXBIO has established partnerships with major biopharmaceutical companies to advance its NAV technology. Further reading. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. Continue following MarketBeat Before you consider REGENXBIO, you'll want to hear this. MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and REGENXBIO wasn't on the list. While REGENXBIO currently has a Moderate Buy rating among analysts, top-rated analysts believe these five stocks are better buys. Nuclear energy is entering a new growth cycle as rising power demand, expanding data centers, and renewed policy support bring the sector back into focus. 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REGENXBIO reported second quarter 2026 financial results, highlighting progress across its gene therapy pipeline. The company received over $200 million in July 2026, including a $100 million milestone payment from AbbVie and approximately $108 million from a public offering, extending its cash runway into Q4 2027. The Phase III AFFINITY DUCHENNE trial for RGX-202 met its primary endpoint with high statistical significance. REGENXBIO plans to initiate a biologics licence application submission in Q3 2026, targeting potential approval in 2H 2027. Second quarter revenues reached $108.0 million, compared to $21.4 million in the prior year period, primarily due to the AbbVie milestone payment. The company reported net income of $22.7 million for the quarter. REGENXBIO expects topline data from pivotal wet AMD studies in Q4 2026 and confirmed plans to resubmit the RGX-121 biologics licence application in Q3 2026.
REGENXBIO Inc. (NASDAQ:RGNX) receives average rating of "Moderate Buy" from analysts. August 4, 2026 Key points. * Analyst sentiment is moderately bullish: Ten of 13 analysts rate REGENXBIO a buy, while two recommend selling and one recommends holding. The average 12-month price target is $23.82, versus a recent share price of $9.96. * Recent financial results fell short of expectations: REGENXBIO reported a quarterly loss of $1.72 per share versus the estimated $1.36 loss, while revenue of $6.39 million was well below the $25.84 million consensus estimate. * Institutional ownership remains high, but insiders have been selling: Institutions and hedge funds own 88.08% of the stock, while company insiders sold 73,803 shares worth approximately $886,937 over the past 90 days. * Five stocks to consider instead of REGENXBIO. Shares of REGENXBIO Inc. (NASDAQ:RGNX - Get Free Report) have received a consensus rating of "Moderate Buy" from the thirteen ratings firms that are covering the stock, Marketbeat.com reports. Two analysts have rated the stock with a sell recommendation, one has assigned a hold recommendation and ten have issued a buy recommendation on the company. The average 12 month price target among analysts that have issued a report on the stock in the last year is $23.8182. Several analysts have issued reports on the company. Barclays cut their price objective on REGENXBIO from $37.00 to $12.00 and set an "overweight" rating on the stock in a research note on Friday, May 15th. Weiss Ratings reiterated a "sell (e+)" rating on shares of REGENXBIO in a report on Monday, June 8th. Leerink Partners upped their price target on REGENXBIO from $17.00 to $18.00 and gave the company an "outperform" rating in a research report on Monday, June 22nd. Zacks Research lowered REGENXBIO from a "hold" rating to a "strong sell" rating in a research report on Tuesday, July 14th. Finally, Robert W. Baird raised their price objective on REGENXBIO from $27.00 to $32.00 and gave the stock an "outperform" rating in a research note on Monday, June 22nd. REGENXBIO stock performance. Shares of REGENXBIO stock opened at $9.96 on Tuesday. The firm has a market cap of $514.93 million, a PE ratio of -1.78 and a beta of 0.94. The firm's 50-day simple moving average is $9.70 and its 200-day simple moving average is $9.49. REGENXBIO has a 1 year low of $5.45 and a 1 year high of $16.19. REGENXBIO (NASDAQ:RGNX - Get Free Report) last posted its quarterly earnings results on Thursday, May 14th. The biotechnology company reported ($1.72) EPS for the quarter, missing analysts' consensus estimates of ($1.36) by ($0.36). The firm had revenue of $6.39 million during the quarter, compared to analyst estimates of $25.84 million. REGENXBIO had a negative return on equity of 232.50% and a negative net margin of 330.23%. On average, equities analysts predict that REGENXBIO will post -3.69 earnings per share for the current year. Insider buying and selling at REGENXBIO. In other REGENXBIO news, insider Steve Pakola sold 2,161 shares of the stock in a transaction on Thursday, July 2nd. The shares were sold at an average price of $13.64, for a total transaction of $29,476.04. Following the sale, the insider directly owned 213,009 shares of the company's stock, valued at approximately $2,905,442.76. This represents a 1.00% decrease in their ownership of the stock. The sale was disclosed in a document filed with the Securities & Exchange Commission, which is available at the SEC website. Insiders have sold 73,803 shares of company stock worth $886,937 over the last 90 days. 14.22% of the stock is currently owned by corporate insiders. Institutional inflows and outflows. Several hedge funds and other institutional investors have recently modified their holdings of RGNX. Versant Capital Management Inc boosted its holdings in shares of REGENXBIO by 60.4% during the second quarter. Versant Capital Management Inc now owns 2,741 shares of the biotechnology company's stock valued at $33,000 after acquiring an additional 1,032 shares during the period. Deutsche Bank AG increased its holdings in shares of REGENXBIO by 0.8% in the 4th quarter. Deutsche Bank AG now owns 192,582 shares of the biotechnology company's stock worth $2,773,000 after acquiring an additional 1,527 shares during the period. Captrust Financial Advisors increased its holdings in shares of REGENXBIO by 13.2% in the 4th quarter. Captrust Financial Advisors now owns 15,228 shares of the biotechnology company's stock worth $219,000 after acquiring an additional 1,781 shares during the period. Diversified Trust Co increased its holdings in shares of REGENXBIO by 15.9% in the 1st quarter. Diversified Trust Co now owns 15,499 shares of the biotechnology company's stock worth $130,000 after acquiring an additional 2,121 shares during the period. Finally, Intech Investment Management LLC lifted its position in REGENXBIO by 12.2% in the 4th quarter. Intech Investment Management LLC now owns 21,367 shares of the biotechnology company's stock valued at $308,000 after purchasing an additional 2,327 shares during the last quarter. 88.08% of the stock is owned by institutional investors and hedge funds. About REGENXBIO. REGENXBIO Inc is a clinical-stage biotechnology company specializing in the development of gene therapies using its proprietary NAV(R) AAV (adeno-associated virus) platform. The company engineers next-generation AAV vectors designed to deliver functional genes to targeted cells, aiming to address a range of rare genetic diseases and ocular, metabolic and neurologic disorders. REGENXBIO's pipeline features several product candidates in various stages of preclinical and clinical development, including RGX-314 for wet age-related macular degeneration, RGX-121 for mucopolysaccharidosis II (Hunter syndrome) and RGX-121 for other rare lysosomal storage diseases. In addition to its internally funded programs, REGENXBIO has established partnerships with major biopharmaceutical companies to advance its NAV technology. This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to [email protected]. Before you consider REGENXBIO, you'll want to hear this. MarketBeat keeps track of Wall Street's top-rated and best performing research analysts and the stocks they recommend to their clients on a daily basis. MarketBeat has identified the five stocks that top analysts are quietly whispering to their clients to buy now before the broader market catches on... and REGENXBIO wasn't on the list. While REGENXBIO currently has a Moderate Buy rating among analysts, top-rated analysts believe these five stocks are better buys. Tesla, Nvidia, and Google helped shape the last era of market growth, but the next wave could come from a new group of companies. Inside this report, you'll find 7 stocks that could play a major role in the next tech-driven market boom.
REGENXBIO has priced an underwritten public offering of 10,003,889 shares of common stock at $9.00 per share and 1,111,111 pre-funded warrants at $8.9999 per warrant. The biotechnology company expects to raise approximately $100 million in gross proceeds before underwriting discounts and commissions. The offering is expected to close on 20 July 2026, subject to customary closing conditions. REGENXBIO has also granted underwriters a 30-day option to purchase an additional 1,667,250 shares at the public offering price. Morgan Stanley, J.P. Morgan, Leerink Partners and Mizuho are acting as joint book-running managers. The company develops gene therapies for rare and retinal diseases, with a pipeline including treatments for Duchenne muscular dystrophy and mucopolysaccharidosis disorders.
REGENXBIO has dosed the first patient in its Phase IIb/III NAAVIGATE trial of surabgene lomparvovec for diabetic retinopathy using suprachoroidal delivery. The milestone triggered a $100 million payment from partner AbbVie. The multicentre, randomised trial will evaluate the investigational gene therapy in subjects with non-proliferative diabetic retinopathy without centre-involved diabetic macular edema. Participants will receive surabgene lomparvovec at 1.0x10¹¹ genome copies per eye, with the primary endpoint measuring two-step improvement on the diabetic retinopathy severity scale at one year. The Phase IIb portion is expected to enrol approximately 135 participants in the United States. REGENXBIO plans to present two-and-a-half-year data from the ALTITUDE trial at the American Society of Retina Specialists meeting in July 2026.