Associate Director - Rare Disease Product Marketing
About the job
- A bachelor's degree is required; an advanced degree such as a Master of Science, Doctor of Philosophy, Master of Business Administration, or Doctor of Pharmacy in life sciences, business, or a healthcare-related field is preferred.
- At least 7 years of product marketing, brand management, marketing strategy, commercial insights, or related commercial experience within the biotechnology or pharmaceutical industry is required.
- Experience supporting or leading product launch preparation and lifecycle activities, including customer segmentation, brand strategy, promotional material development and approval, and field enablement, is required.
- A strong understanding of drug development and commercialization processes is required, ideally within a rare disease, specialty, or gene therapy setting.
- Experience integrating primary qualitative and quantitative research and secondary research into strategic recommendations, value propositions, and marketing tactics is required.
- Experience developing healthcare professional, caregiver, and payer-facing content and working through promotional review and approval processes while maintaining scientific accuracy and compliance is required.
- Experience collaborating cross-functionally with marketing, medical, market access, regulatory/legal, commercial operations, and field teams is required.
- Strong analytical and problem-solving skills and the ability to operate in ambiguity are required.
- Proficiency with Excel, PowerPoint, and relevant market research, customer, and analytics tools is required.
- Experience managing agencies, vendors, and external partners to deliver marketing and insights work on time and within budget is required.
- Lead the development and execution of integrated rare disease brand plans, including customer segmentation, positioning, messaging, and omnichannel tactics for healthcare professional, payer, caregiver, and field audiences.
- Translate market research, competitive intelligence, and field feedback into recommendations that inform brand planning, launch readiness, value positioning, and marketing performance.
- Develop and manage healthcare professional- and payer-facing promotional materials, including digital and field-facing tools, through the promotional review process to ensure strategic alignment, scientific accuracy, and timely execution.
- Manage agency and vendor partnerships for market research, creative development, media, analytics, and other marketing services to ensure high-quality deliverables on time and within budget.
- Lead annual operating planning and marketing budget management; prioritize initiatives, track spend, and coordinate promotional asset lifecycle updates.
- Collaborate with Commercial, Medical, Market Access, Regulatory/Legal review, Research and Development, Corporate Affairs, and field teams to align brand strategy, customer engagement, advisory boards, peer-to-peer education, and congress initiatives.
- Communicate actionable recommendations to senior stakeholders and partner with commercial training and field teams to enable rollout and adoption of new materials and initiatives.
- Travel up to 25% for medical congresses, conferences, and internal meetings.
- Rare disease and/or Duchenne experience is preferred.
- Advanced degree in life sciences, business, or a healthcare-related field is preferred.
- Experience in a rare disease, specialty, or gene therapy setting is preferred.
- Rare disease experience in agency, vendor, or external-partner management is strongly preferred.
About the company
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
- Q2 2026 revenue hit $108.0 million after AbbVie's $100 million milestone payment.
- RGX-202 met its primary endpoint, with BLA initiation targeted for Q3 2026.
- Cash reached about $313 million pro forma, funding operations into Q4 2027.
What critics are saying
- FDA's August 2026 clinical hold on RGX-121 blocks resubmission and delays Hunter syndrome revenue.
- September 2026 Sarepta settlement surrendered AAVrh74 claims, weakening future ELEVIDYS leverage.
- January 2026 Zolgensma patent expiry in the United States erodes the legacy royalty base.
What makes REGENXBIO unique
- NAV platform owns 100-plus AAV vectors, powering royalties and internal programs since 2009.
- AbbVie's August 2026 NAAVIGATE milestone validated RGX-314 across wet AMD and diabetic retinopathy.
- Novartis royalties still fund operations despite January 2026 U.S. Zolgensma patent expiry.
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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
1%2 year growth
0%REGENXBIO's diabetic eye gene therapy reaches three-year mark. Six of ten patients in a small trial improved by two or more steps on a severity scale after one treatment, though the data are interim. Most treatments for diabetic eye disease ask patients to come back again and again for injections. REGENXBIO is testing whether one visit could do the job for years, and its latest numbers, presented at the Retina Society's 59th Annual Scientific Meeting in Los Angeles, are worth a careful read [1]. The company reported three-year follow-up data (as of 17 August 2026) from its Phase II ALTITUDE trial of surabgene lomparvovec, or sura-vec (also known as ABBV-RGX-314, developed in collaboration with AbbVie). Participants had non-proliferative diabetic retinopathy, the earlier stage of the disease, before abnormal blood vessels start growing in the eye. Sura-vec is a gene therapy. Doctors deliver it in a single in-office procedure, and it uses a viral vector (a modified virus that works as a delivery vehicle) to carry instructions into cells at the back of the eye. Those cells then make a fragment of an antibody that blocks VEGF, a protein that encourages new, leaky blood vessels to form and fluid to build up in the retina. Today's anti-VEGF injections do a similar job from the outside, which is why they have to be repeated. The gene therapy is meant to turn the eye into its own supplier. For the patients in this analysis, the treatment came with a short course of steroid eye drops to head off inflammation. Among participants who received Dose Level 3 and had three-year visits, 60% improved by two or more steps on the Diabetic Retinopathy Severity Scale (DRSS), the grading system eye doctors use to rate how advanced the disease is. That's six out of ten people. None of them needed additional treatment for diabetic retinopathy, and none had vision-threatening events. There's a second finding. Four participants had improved by one step at year one without extra anti-VEGF injections, and three of those four went on to reach a two-step improvement by year three, again without further treatment. Across 17 participants followed through three years, the company saw no intraocular inflammation and no new safety signals related to sura-vec. Dr Steve Pakola, chief medical officer of REGENXBIO, called the results "highly encouraging," and said the durability "strengthens our conviction in sura-vec as a potentially transformative, one-time treatment." Ten patients is a very small group. The company also says these are interim results from analyses it performed itself, in an ongoing trial. And 60% still means four of the ten didn't hit the two-step mark, though the release doesn't explain what happened with them. There's a footnote worth knowing about, too. One participant who reached a two-step improvement at year two later received two supplemental anti-VEGF injections. Their investigator chose to treat the study eye at the same time as the other eye, which needed treatment for swelling in the center of the retina, even though the study eye showed no sign of progression. That participant was then lost to follow-up and has no year-three data. It's a reminder of how messy real patients are, and how a tidy percentage can sit on top of judgment calls. The dose is being carried forward regardless. Dose Level 3 is now being tested in the larger Phase IIb/III NAAVIGATE trial in the same patient group. Dr Nikolas London, a retina specialist at Retina Consultants San Diego and an ALTITUDE investigator, explained why the goal matters. "For patients with non-proliferative diabetic retinopathy, the goal is to intervene before the disease progresses to vision-threatening complications and irreversible vision loss," he said. "However, current preventive treatment requires repeat injections, which is a significant burden for a population that is largely working-age adults." That burden isn't small. Diabetic retinopathy is the leading cause of vision loss in adults between 24 and 75 worldwide, and it affects nearly 10 million people in the United States alone. For patients at the earlier stage, the current options are typically "watchful waiting" or anti-VEGF treatment. The longevity angle. Diabetes wears on the body slowly, and the eye is one of the places it shows first. Losing sight in midlife or later means losing independence, work, driving, reading, the little things that make a long life feel like one worth having. A single treatment that could hold off that decline would fit neatly into the longevity field's growing interest in one-time therapies for chronic problems, rather than lifelong maintenance. Whether sura-vec earns that place depends on bigger trials. For now it's an early, small, carefully hopeful signal. LTUI: 2865 Regenxbio. Powered by: Longevity level(s): * Level 5: Aging disease management * Level 7: Targeted aging disease reversal Overview. Regenxbio is a clinical-stage biotechnology company focused on developing adeno-associated virus (AAV) gene therapies designed to deliver functional genes to cells for the treatment of genetic defects and rare diseases. The company's proprietary NAV(R) Technology Platform enables targeted delivery of therapeutic genes to address underlying disease causes across retinal, metabolic, and neurodegenerative therapeutic areas. Through single-administration treatments intended to provide long-lasting effects, Regenxbio aims to significantly alter disease progression and improve patient outcomes for individuals with serious unmet medical needs. Access all data points on DLT: * Financial Data * Patents * Technology * Management * Advisory Board * News * Classifications * Similar Companies
Beam sues 2 biotechs, alleging theft of base editing trade secrets. Beam claimed a former scientist stole confidential information and quietly spun up a rival rare disease treatment at YolTech Therapeutics. Serapha Bio, which licensed that therapy, denies the charges. Published Sept. 29, 2026 Beam Therapeutics is suing the Chinese biotechnology company YolTech Therapeutics and its U.S. partner Serapha Bio, alleging that an experimental treatment licensed between the two companies was developed using trade secrets stolen from Beam. In a complaint filed Sept. 25 with the U.S. District Court in Massachusetts, Beam alleged that a former employee, Zi Jun "Emma" Wang, "secretly founded a Chinese biotechnology company called YolTech while still working at Beam." Five months after leaving the gene editing company, Wang "filed patent applications on behalf of YolTech based on Beam's confidential and trade secret information." Now, Beam is seeking court intervention to "stop the misuse of its trade secrets" and "recover the value" that YolTech and Serapha "unlawfully extracted from Beam's investments." "Serapha categorically refutes Beam's claims and will vigorously defend the company," Shiva Fritsche, Serapha's chief corporate affairs officer, wrote in an email to BioPharma Dive on Tuesday. In its complaint, Beam claimed that, while working as a scientist at Beam from 2021 to 2022, Wang started a company in China that would become YolTech. The Massachusetts biotech accused Wang of accessing data "in a manner that departed sharply from her ordinary work, retrieving and reviewing dozens of Beam records she had never opened before, across technical areas well beyond her own, including at night and on weekends." Wang did not disclose that she intended to leave and create a similar biotech, Beam claimed. The complaint also names YolTech's CEO, Yuxuan "Jensen" Wu, as a defendant. YolTech then struck a deal with Serapha Bio, handing off rights to an experimental program for the rare disease alpha-1 antitrypsin deficiency, or AATD - a common target for gene editing biotechs - for an undisclosed upfront payment. In June, Serapha announced plans to quickly go public through a reverse merger deal and develop that therapy. That effort was supported by $230 million in funding from investors including RA Capital Management and RTW Investments. Like Beam's other therapies, the AATD treatment BEAM-302 is built on "base editing" technology that precisely changes a specific nucleotide, or letter, in genetic code. In clinical testing so far, BEAM-302 has helped trial participants produce a key protective protein they lack. The company has begun enrolling participants in a new study cohort. If the results are positive, Beam will pursue an accelerated U.S. approval. Serapha's therapy, codenamed "SERP-01," targets the same mutation and changes a nucleotide at the same place in DNA as BEAM-302. Beam argued in its court filing that YolTech's treatment is made with the help of proprietary technologies originally developed by the Massachusetts company. Serapha in mid-September named a slate of executives tasked with shepherding its AATD program through clinical testing. Those hires include new CEO Kenneth Mills, the former leader of Regenxbio; and Chief Medical Officer Weston Miller, a veteran gene editing executive who'd previously worked at biotechs such as Epicrispr Technologies and Graphite Bio. Serapha aims to start a Phase 2/3 trial of SERP-01 later this year or in early 2027.
REGENXBIO pauses RGX-121 FDA review. * The FDA placed a clinical hold on REGENXBIO's RGX-121 gene therapy program after spine MRI findings in five CAMPSIITE study participants. * REGENXBIO stated that affected participants remain clinically well, with stable or improved neurocognitive and neurobehavioral assessments. * The company does not expect to resubmit the RGX-121 BLA in the near term and will evaluate additional data with regulators. REGENXBIO (NASDAQ:RGNX) reported that the U.S. Food and Drug Administration placed a clinical hold on its investigational MPS II gene therapy RGX-121 following spine MRI findings in five participants in the CAMPSIITE study. The findings involved small spinal nodules or cystic masses identified in participants who received RGX-121 through intracisternal or intraventricular dosing approximately three to six years ago. REGENXBIO stated that investigators considered the findings nonserious and radiologists believe the abnormalities are likely benign, while no brain nodules or masses were observed. The company stated that it does not expect to resubmit the RGX-121 Biologics License Application in the near term. REGENXBIO and partner NS Pharma are reviewing additional imaging and long-term follow-up data while incorporating FDA feedback into future plans. The company continues advancing other programs, including a planned Duchenne gene therapy BLA submission in the current quarter and wet age-related macular degeneration pivotal trial topline data expected in the fourth quarter. Frequently asked questions. RGNX signals
RBC Capital Downgrades EyePoint Pharmaceuticals (NASDAQ:EYPT) Amid Mixed DURAVYU trial results for wet AMD. Aug 18, 2026 Market News FMPRBC Capital Downgrades EyePoint Pharmaceuticals (NASDAQ:EYPT) Amid Mixed DURAVYU... * RBC Capital downgraded EyePoint Pharmaceuticals (NASDAQ:EYPT) due to mixed Phase 3 trial results for its wet AMD drug, DURAVYU. * The LUGANO trial for DURAVYU missed its primary endpoint, influenced by a small patient subgroup, despite showing comparable efficacy to standard treatment and a 42% reduction in treatment burden in a separate analysis. * EyePoint's stock plummeted over 66.98% to a 52-week low of $3.95, further pressured by positive trial data from competitor REGENXBIO (NASDAQ:RGNX) for its own wet AMD treatment. RBC Capital downgrades its rating on EyePoint Pharmaceuticals to Sector Perform from a previous Outperform rating. EyePoint is a pharmaceutical company that develops treatments for serious eye disorders. The downgrade occurs as the stock price is $4.87, following a significant drop related to recent company news. The change in rating follows the release of mixed results from EyePoint's Phase 3 LUGANO clinical trial for its drug, DURAVYU. This trial tested the drug's effectiveness in treating wet age-related macular degeneration (AMD), a common cause of vision loss. As highlighted by Reuters, the study did not meet its main goal. The trial's primary objective was not met due to a small group of patients. This group, representing 4% of participants, had vision loss from causes unrelated to wet AMD. This unexpected outcome affected the overall data for the study's main test, preventing it from showing a clear benefit on vision. However, a separate analysis that excluded this small group showed DURAVYU performed as well as the current standard treatment, aflibercept. The trial also showed other positive results, including a 42% reduction in treatment burden. This means patients using DURAVYU required fewer treatments over time compared to the standard drug. The market reacted strongly to the news, with EyePoint shares falling 66.98% to a new 52-week low of $3.95 during the day. At the same time, a competitor, REGENXBIO, announced positive trial data for its own wet AMD treatment, adding pressure to the competitive environment for EyePoint. Market news and analyst rating coverage Rayan Ahmad covers market news, analyst rating changes, and company developments for the FMP blog. His work focuses on summarizing price-target updates, earnings results, and broker actions into accessible, data-backed market updates. Financial data for every need. Real-time quotes and 30+ years of historical data, including prices, fundamentals, and insider transactions - all accessible via API. Stock Screener 2017-2026 (C) FMP
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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