A

AskBio

Clinical-stage gene therapy company developing AAVs

Intern IT - Enterprise Architecture

Summer 2026Updated Deadline 3/20/26
No salary listed
Internship
Bachelor's
Philadelphia, PA, USA+1 moreMore locations: Research Triangle, Durham, NC, USA
In Person
Data & AnalyticsData Management

About the job

Requirements
  • Must be pursuing a Bachelor’s degree in Information Systems, Computer Science, Data Analytics, Business Analytics, Management Information Systems (MIS) or related discipline
  • Must be at least a Junior at the time of application
  • Required Systems and Tool experience: Comfort navigating documentation repositories (SharePoint, Confluence, or similar); Ability to learn new internal systems (EA Repository/toolset, portfolio tools) with guidance; Experience organizing information in structured templates or spreadsheets
  • Required technical and analytical skills: Ability to work with Excel or Google Sheets to organize, filter, clean, and compare simple datasets; Coursework exposure to data models, databases, or information architecture concepts; Ability to understand and document metadata such as system names, owners, lifecycle stage, integration, and purpose; Familiarity with basic data quality concepts (completeness, consistency, duplication, accuracy)
  • Strong sense of accountability and ability to prioritize multiple tasks
  • Excellent organizational skills and meticulous attention to detail
  • Flexible and willing to support a variety of tasks for the IT team
  • Proactive, solution‑driven mindset with strong written and verbal communication skills
  • Available to work full‑time at the office during the summer months (May–August)
Responsibilities
  • Support updates to the Enterprise Architecture (EA) repository, focusing on improving accuracy, completeness, and consistency of application‑related data
  • Learn how EA data supports key IT functions including portfolio management, rationalization, capability mapping, integrations, and lifecycle planning
  • Review existing metadata and documentation for systems, applications, interfaces, owners, lifecycle stage, technical stacks, and business capabilities served
  • Partner with Application Owners, IT SMEs, and Business Partners to validate and correct EA repository entries
  • Observe how the EA repository is used for road mapping, budgeting, and strategic planning
  • Review application health, risk, and cost attributes (as provided) to ensure logical alignment across the portfolio
  • Participate in EA governance activities, including standardizing data definitions and improving intake/update processes
  • Assist in developing diagrams, portfolios, and dashboards derived from EA data
  • Maintain records of changes made in the EA repository and track open items requiring stakeholder follow‑up
  • Communicate findings clearly through summaries, visuals, and status updates
  • Collect insights and recommendations to support the creation of an EA Data Quality Playbook
  • Present a final intern presentation summarizing accomplishments, insights, and proposed next steps

About the company

A

AskBio

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Website

AskBio focuses on developing adeno-associated virus (AAV) gene therapies for genetic and complex disorders. Therapies use AAV vectors to deliver therapeutic DNA into patients’ cells, advancing from research and preclinical work through clinical trials toward commercialization. Its differentiation comes from a clinical-stage portfolio, in-house R&D, and partnerships that emphasize collaboration and shared development. Its goal is to bring gene therapies to patients with unmet medical needs by validating therapies clinically and pursuing strategic collaborations for commercialization.

Company Size

501-1,000

Company Stage

Acquired

Total Funding

$239.1M

Headquarters

Durham, NC

Founded

2001

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Simplify's Take

What believers are saying

  • AB-1009 dosed its first patient on May 11, 2026, de-risking Pompe execution.
  • AB-1002 finished enrollment April 1, 2026, with first results expected in first-half 2027.
  • AskBio presented six ASGCT 2026 updates and advanced Viralgen commercial-scale supply.

What critics are saying

  • AB-1009 and AB-1002 remain unproven; failures in 2027 erase most pipeline value.
  • Pompe licensing from Genethon adds royalty and control pressure if data disappoints.
  • Bayer can reprioritize AskBio after broader group pressure, ending standalone gene-therapy investment.

What makes AskBio unique

  • Bayer-owned AskBio runs integrated AAV discovery, development, and Viralgen manufacturing in-house.
  • AB-1009 licensed Genethon’s patented component, strengthening Pompe disease IP and global freedom-to-operate.
  • AskBio’s 173-patient GenePHIT and 2026 REGENERATE-PD manufacturing scale show rare late-stage depth.

Help us improve and share your feedback! Did you find this helpful?

Benefits

  • Health Insurance
  • Dental Insurance
  • Vision Insurance
  • Life Insurance
  • Disability Insurance
  • Health Savings Account/Flexible Spending Account
  • Unlimited Paid Time Off
  • Flexible Work Hours
  • Remote Work Options
  • Paid Vacation
  • Paid Sick Leave
  • Paid Holidays

Growth & Insights and Company News

6 month growth

3%

1 year growth

0%

2 year growth

1%
AskBio
Oct 22nd, 2026
AskBio announces 5 presentations at the European Society of Gene and Cell Therapy (ESGCT) 33rd Annual Congress.

AskBio announces 5 presentations at the European Society of Gene and Cell Therapy (ESGCT) 33rd Annual Congress. * Five poster presentations will highlight exciting progress in AskBio's adeno-associated virus (AAV) manufacturing technologies Durham, N.C., - October 22, 2026 - AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, and its subsidiary Viralgen Vector Core S.L.U. (Viralgen) will deliverfivepresentations offering insights into advancements in manufacturing technologies of adeno-associated virus (AAV) therapies for a range of diseases, at the European Society of Gene and Cell Therapy (ESGCT) 33rd Annual Congress taking place October 27-30, 2026, in Hamburg, Germany. "ESGCT is one of the defining scientific meetings for the global cell and gene therapy community, and at AskBio we are proud to continue contributing to the science that is shaping the future of these potentially transformative medicines," said Gustavo Pesquin, Chief Executive Officer, AskBio. "Our presence this year highlights advances across our AAV manufacturing platform and our work to make production increasingly reproducible, measurable and robust. Collectively, these presentations reflect our continued investment in the technologies, analytical capabilities and process understanding needed to consistently and reliably manufacture gene therapies at scale with a goal to ultimately bring them to the patients who may benefit." AskBio's presentations include (all times CET): * Development of a stable producer cell line for successful AAV production (Poster #P0767). Poster Session I, Wednesday, October 28, 1:30-3:30 p.m., Elena Lazarova, Scientist II, Cell Line Development, AskBio. * In-line capacitance probes enable enhanced and consistent high-quality rAAV manufacturing (Poster #P0803). Poster Session I, Wednesday, October 28, 1:30-3:30 p.m., Andoni Gonzalez, R&D Associate Scientist, Viralgen. * Optimization of recombinant adeno-associated virus vector production: Integrating synthetic molecules to enhance vector yield and quality for clinical gene therapy applications (Poster #P0801). Poster Session I, Wednesday, October 28, 1:30-3:30 p.m., Jose Luis Abad Minguez, R&D Senior Scientist, Viralgen. * Repeatability and intermediate precision of a genome integrity assay for rAAV by long-read sequencing (Poster #P0253). Poster Session I, Wednesday, October 28, 1:30-3:30 p.m., Oihana Aldasoro, R&D Associate Scientist, Viralgen. * Investigations of AAV stability under stress conditions using small-scale models (Poster #P0802). Poster Session II, Thursday, October 29, 2:00-4:00 p.m., Ferran Vila Julia, MSAT Scientist, Viralgen. AskBio continues to develop an ambitious portfolio of investigational AAV-based gene therapies with the potential to treat some of the world's most debilitating diseases, including congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson's disease, and Pompe disease. By targeting diseases in several therapeutic areas, AskBio aims to deliver innovative treatments that may possibly benefit more than 35 million patients worldwide.[1-5] About AskBio AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to steering gene therapy into a new era where it can transform the lives of a wider range of people living with rare and more common diseases. The company maintains a portfolio of clinical programs across a range of disease indications related to a single gene or multiple factors across cardiovascular, central nervous system, and neuromuscular conditions, with a clinical-stage pipeline that includes investigational therapeutics for heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson's disease, and Pompe disease. AskBio's end-to-end gene therapy platform includes its Pro10(TM) technology and Aava(TM) manufacturing platform, which make gene therapies more accessible by making research and commercial grade manufacturing more affordable. With global headquarters in Durham, North Carolina, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field with over 900 employees in five countries, the company holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at www.askbio.com or follow AskBio Inc. on LinkedIn. About Viralgen Viralgen Vector Core S.L.U. is a leading Contract Development and Manufacturing Organization (CDMO) specializing exclusively in adeno-associated virus (AAV)-based cell and gene therapies. Founded in 2017 as a subsidiary of AskBio Inc. within the Bayer AG group, Viralgen delivers end-to-end support from early development to large-scale commercial manufacturing. Leveraging their proprietary Pro10(TM) suspension cell line and Aava(TM) manufacturing platform, Viralgen achieves industry-leading, high-yield, scalable manufacturing across all AAV serotypes. Located in San Sebastián, Spain, its state-of-the-art facility includes three cGMP suites with 2,000-liter capacities each, certified by the AEMPS/EMA, and integrates services including plasmid production, process optimization, fill-finish, and in-house QC testing. With over 1,500 AAV batches produced, Viralgen provides reliable, efficient solutions to bringing life-changing gene therapies to patients with greater speed and consistency. Learn more at https://viralgen.com and follow AskBio Inc. onLinkedIn. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, "Health for all, Hunger for none," the company's products and services are designed to support efforts to master major challenges presented by a growing and aging global population and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2025, the Group employed around 88,000 people and had sales of 45.6 billion euros. R&D expenses amounted to 5.8 billion euros. For more information, go to www.bayer.com. AskBio Forward-Looking Statements This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible," and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding AskBio's clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with its third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio's business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. Media Contact: Phil McNamara Vice President, Corporate Communications, AskBio E: [email protected] T +1 984.520.7211 References * Parkinson's Foundation. Understanding Parkinson's. Statistics. Available at: https://www.parkinson.org/understanding-parkinsons/statistics. Accessed: October 2026. * Malik A, et al. Congestive Heart Failure. In: StatPearls. Treasure Island (FL): StatPearls Publishing; Last update February 2025. * MedlinePlus Genetics. NIH. Limb-girdle muscular dystrophy. Available at: https://medlineplus.gov/genetics/condition/limb-girdle-muscular-dystrophy/#frequency. Accessed October 2026. * Goh Y, et al. Multiple system atrophy [published online ahead of print, 2023 Mar 16]. Pract Neurol. 2023; practneurol-2020-002797. * United Pompe Foundation. About Pompe Disease. Available at: https://www.unitedpompe.com/about-pompe-disease/. Accessed: October 2026.

PR Newswire
Jul 27th, 2026
The Speak Foundation announces 2026 LGMD Scientific Summit.

The Speak Foundation announces 2026 LGMD Scientific Summit. Jul 27, 2026, 10:00 ET Global researchers, clinicians, regulators, biotechnology leaders, and patient advocates will convene to advance LGMD research and therapeutic development WASHINGTON, July 27, 2026 /PRNewswire/ - The Speak Foundation today announced the 2026 LGMD Scientific Summit, an international scientific meeting that will convene leading researchers, clinicians, biotechnology innovators, regulators, and patient advocates to discuss recent advances in Limb-Girdle Muscular Dystrophy (LGMD) research, clinical development, and therapeutic innovation. The virtual event will be held July 31, 2026. The Summit comes at a pivotal moment for the LGMD field as multiple therapeutic programs advance through clinical development, new biomarkers emerge, and innovative clinical trial approaches continue to reshape rare disease research. Scientific sessions will highlight gene therapy, regenerative medicine, biomarker qualification, natural history studies, patient-focused drug development, and collaborative strategies designed to accelerate new treatments for individuals living with LGMD. FEATURED SCIENTIFIC LEADERS Featured researchers include Douglas Sproule, MD, MSc (BridgeBio Neuromuscular); Louise Rodino-Klapac, PhD (Sarepta Therapeutics); Barry Byrne, MD, PhD (University of Florida); John Vissing, MD, DMSci (University of Copenhagen); Nicholas Johnson, MD, MSCI, FAAN (Virginia Commonwealth University); Peter Kang, MD (University of Minnesota); Tahseen Mozaffar, MD, FAAN (University of California, Irvine); Simone Spüler, MD (Charité - Universitätsmedizin Berlin); Brad Williams, PhD (Jain Foundation), and Elizabeth McNally, MD, PhD (Northwestern University). Additional scientific and clinical presentations will be delivered by representatives from biotechnology companies and nonprofit research organizations advancing therapies for LGMD, including BridgeBio Neuromuscular, Sarepta Therapeutics, AskBio, Genethon, MyoPax, Edgewise Therapeutics, Advertent Biotherapeutics, Kinea Bio, Myogenica, and Cure Rare Disease. PATIENT-CENTERED DRUG DEVELOPMENT A featured panel will bring together representatives from the U.S. Food and Drug Administration and the patient community to examine patient-centered clinical trial design, stakeholder engagement, and collaborative approaches to accelerating therapeutic development for rare neuromuscular diseases. SCIENTIFIC PERSPECTIVES Nicholas Johnson, MD, MSCI, FAAN Professor and Executive Vice Chair, Department of Neurology Virginia Commonwealth University "We have entered a transformative period for LGMD research. Scientific advances are creating opportunities that were unimaginable only a decade ago, but realizing their full potential will require continued collaboration, rigorous science, and a shared commitment to ensuring that no LGMD subtype is left behind." Douglas Sproule, MD, MSc Chief Medical Officer, BridgeBio Neuromuscular "We are deeply grateful to The Speak Foundation for convening the LGMD community and fostering collaboration among patients, caregivers, clinicians, researchers, and industry, while ensuring the patient perspective remains central to scientific progress. We also thank the individuals and families who have participated in our clinical studies and entrusted us with their time and hope. It is a privilege to contribute to advancing therapies for LGMD, and that trust reinforces our commitment to rigorous science and the urgent development of meaningful new treatments." The Speak Foundation gratefully acknowledges BridgeBio Neuromuscular, Platinum Sponsor of the 2026 LGMD Scientific Summit, and AskBio, Bronze Sponsor, for their support and commitment to advancing LGMD research and therapeutic development. Registration is open to researchers, clinicians, biotechnology professionals, regulators, patients, caregivers, and members of the global LGMD community. To view the program and register, visit TheSpeakFoundation.com/LGMD-Summit. ABOUT THE SPEAK FOUNDATION Founded in 2008, The Speak Foundation is a patient-led nonprofit organization dedicated to improving the lives of individuals and families affected by Limb-Girdle Muscular Dystrophy through advocacy, research, education, and public policy. The Foundation convenes the global LGMD community by bringing together patients, researchers, clinicians, industry, regulators, and policymakers to accelerate research, improve access to specialized care, and advance therapeutic development. Signature initiatives include the International LGMD Conference, LGMD Scientific Summit, LGMD Scientific Workshop, LGMD Day on the Hill, LGMD News Magazine, and the LGMD Centers of Excellence. SOURCE The Speak Foundation

BioPharm International
May 15th, 2026
AskBio doses first patient in Phase 1/2 gene therapy trial for late-onset Pompe disease.

AskBio doses first patient in Phase 1/2 gene therapy trial for late-onset Pompe disease. AskBio has dosed the first participant in a Phase 1/2 clinical trial evaluating AB-1009, an investigational AAV-based gene therapy for late-onset Pompe disease. AskBio has dosed the first participant in PROGRESS-GT LOPD, a Phase 1/Phase 2 clinical trial evaluating investigational gene therapy AB-1009 for the treatment of late-onset Pompe disease (LOPD).[1] The milestone advances the company's efforts to develop a potentially durable gene therapy approach for the rare neuromuscular disorder, which continues to present significant unmet medical needs despite the availability of enzyme replacement therapies (ERTs). AB-1009 is an adeno-associated virus (AAV)-based investigational gene therapy designed to enable sustained production of acid alpha-glucosidase (GAA), the enzyme deficient in patients with Pompe disease.[1] The therapy is being developed by AskBio, a wholly owned and independently operated subsidiary of Bayer AG.[1] The open-label PROGRESS-GT LOPD trial is expected to enroll approximately 12 adult participants across sites in the United States.[2] Investigators will evaluate the therapy's safety, tolerability, and preliminary efficacy in patients with LOPD.[2] Addressing long-term challenges in Pompe disease. Pompe disease is a rare inherited lysosomal storage disorder caused by mutations affecting production of GAA, leading to glycogen accumulation in muscle cells.[3] In LOPD, patients typically experience progressive skeletal muscle weakness and respiratory decline that worsens over time.[3] "Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical." - Tahseen Mozaffar, MD Although multiple recombinant human GAA enzyme replacement therapies have been approved, clinical limitations remain.[1] Some patients experience declining therapeutic response over time, requiring ongoing infusions and increasing treatment burden.[4] Researchers have increasingly explored gene therapy approaches as a potential strategy for sustained enzyme production and reduced dependence on chronic ERT administration. "Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical," said Tahseen Mozaffar, MD, director of the UCI Health ALS & Neuromuscular Center and principal investigator for the AB-1009 clinical program, in the company announcement.[1] According to AskBio, AB-1009 is designed to address the underlying genetic cause of Pompe disease by enabling continuous endogenous production of GAA following a single administration.[1] Regulatory momentum supports program development. The investigational therapy has already received both FDA Fast Track and Orphan Drug designations, reflecting regulatory recognition of the significant unmet need in Pompe disease.[1] The FDA also accepted the investigational new drug application for AB-1009 earlier this year.[1] Fast Track designation is intended to facilitate development and expedite review of therapies targeting serious conditions with limited treatment options.[5] Orphan Drug designation may provide incentives including market exclusivity, tax credits, and regulatory support for therapies addressing rare diseases.[6] "Reaching this milestone reflects the momentum behind our gene therapy platform and our commitment to working toward improving quality of life for people with rare diseases," said Canwen Jiang, MD, PhD, chief development officer and chief medical officer at AskBio, in the company statement.[1] The clinical advancement also highlights continued investment in neuromuscular gene therapies, an area that has seen substantial growth across both biotech and pharmaceutical sectors over the past several years. Gene therapy continues expanding in rare disease. AAV-based gene therapies have emerged as a major area of focus in rare genetic disease research, particularly for neuromuscular disorders caused by single-gene mutations.[7] Developers are increasingly targeting diseases such as Duchenne muscular dystrophy, limb-girdle muscular dystrophy, spinal muscular atrophy, and Pompe disease using viral vector platforms intended to provide long-term protein expression after a single treatment.[7] Despite growing momentum, challenges remain, including durability of expression, immune responses, manufacturing complexity, and long-term safety monitoring.[7] Still, industry investment continues to accelerate as companies pursue therapies capable of transforming chronic rare diseases into potentially manageable conditions with one-time administration approaches. For patients with LOPD, the initiation of the PROGRESS-GT LOPD study represents another step toward evaluating whether gene therapy may offer a more durable alternative to lifelong enzyme replacement treatment.

Pharma Press Publishing
Mar 25th, 2026
a-Klotho cell and gene therapy potential.

a-Klotho cell and gene therapy potential. 25 March 2026 Avaí Bio recently announced that it has initiated manufacturing of a master cell bank of genetically modified cells that overexpress the α-Klotho protein, working alongside joint venture partner Austrianova through their shared entity, Klothonova. A Master Cell Bank isn't a research milestone. It's a manufacturing milestone. It's the GMP-compliant, fully characterized starting material from which all working cell banks and final therapeutic products will be produced. It's the moment a cell therapy programme transitions from "we think this could work" to "we're building the production infrastructure to deliver it." There is a key 'master' protein in your body called α-Klotho. The scientific community has been quietly obsessed with it since a Japanese researcher named Makoto Kuro-O discovered in 1997 that mice without the Klotho gene aged rapidly and died young - and mice with extra Klotho lived 30% longer. Since then, the research has only gotten more compelling. Peer-reviewed studies have linked higher Klotho levels to reduced risk of Alzheimer's, certain cancers, cardiovascular disease, and kidney failure. Mayo Clinic research has connected declining Klotho to arterial stiffness and vascular calcification. And here's the part that makes this personal: natural α-Klotho levels drop by approximately 50% after age 40. The protein that protects your brain, your heart, and your kidneys starts disappearing right when you need it most. The question the biotech industry is now racing to answer is simple: can you put it back? The global cell therapy market has surpassed $8.2 billion in 2026. The broader cell and gene therapy sector is forecast to surge from $10.4 billion to more than $45 billion by 2035. And at the intersection of regenerative medicine and longevity science, a small biotechnology company just hit a manufacturing milestone that turns the Klotho thesis from laboratory theory into production reality. The Cell-in-a-Box(R) technology is the delivery mechanism that makes the whole approach viable. When you transplant therapeutic cells into a patient, the immune system typically destroys them within days. Austrianova's encapsulation technology protects the cells inside a porous capsule, allowing the cells to continuously produce and secrete the target protein - in this case, α-Klotho - while protecting the cells from immune rejection. It's backed by over 50 peer-reviewed publications and decades of development. Avaí Bio's dual-programme approach targets both the Klothonova anti-aging platform and the Insulinova diabetes program, each leveraging the same Cell-in-a-Box(R) encapsulation technology. The addressable markets are enormous: Alzheimer's disease alone is projected to reach $32.8 billion by 2033, cardiovascular disease remains the leading cause of death globally, and kidney disease affects 850 million people worldwide.

Lifescience Intellipedia Pvt. Ltd.
Apr 3rd, 2025
Bayer Heralds Pipeline Progress, Blockbuster Launches and Advanced

Through AskBio, a wholly owned subsidiary of Bayer, it is developing a gene therapy as a potential new approach for treating congestive heart failure.

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