• Skip to primary navigation
  • Skip to main content
  • Skip to footer
  • Skip to investors navigation
Insmed

Insmed

Powered by Purpose

LinkedIn YouTube Contact Us Job Opportunities

  • Culture
    • Our Culture
      • Mission & Vision
      • Careers

    Our Global Company

    Find available career opportunities in the U.S., Japan, or Europe.

    Find Jobs

  • Patients
    • Our Patients
      • Disease Areas
      • Resources
      • Patient Access

    Current Clinical Trials

    You may be eligible for one of our clinical trials.

    See if you may qualify

  • Science
    • Our Science
      • Our Pillars
        • ARIKAYCE®
        • Brensocatib
        • TPIP
        • Early-Stage Research
      • Pipeline
      • Clinical Trials
      • Investigator-Initiated Research
      • Grants & Funding
    Discovery Dialogues at Insmed

    Discovery Dialogues at Insmed

    Sit down with members of Insmed’s global Research team as they discuss our approach to developing life-transforming medicines while tackling some of the most pressing scientific challenges today.

    Watch the videos

  • Responsibility
    • Our Responsibility
      • Our Commitments
      • Inclusion at Insmed
      • Global Healthcare Compliance
      • Policies and Disclosures

    Responsibility Report

    Our Responsibility Report reflects the culture and purpose we live by every day. We are committed to reporting annually on our progress and commitments.

    Learn More

  • About
    • About Insmed
    • Management Team
    • Board of Directors
    • Partnerships
  • Investors
    • Investors
    • News Releases
    • Events & Presentations
    • Earnings Results
    • Filings
      • Annual Reports
      • SEC Filings
    • Corporate Governance
    • Responsibility Reports
    • Stock Information
    • Investor FAQs
    • Analyst Coverage
    • Email Alerts
Home/Science/Our Pillars/Early-Stage Research

Early-Stage Research

We’re advancing a research engine encompassing a wide range of technologies and modalities, including artificial intelligence-driven protein engineering, gene therapy, and protein manufacturing. By pushing the boundaries of what’s possible within these areas, our Research teams across San Diego, New Hampshire, New Jersey, and Cambridge, UK aim to address numerous challenges facing the development of novel gene therapies and protein therapeutics. Their efforts will fuel our pipeline for years to come, enabling us to develop new and cutting-edge technologies that may address a broad range of rare diseases across therapeutic areas.

Discovery Dialogues at Insmed

Sit down with members of Insmed’s global Research team as they discuss our approach to developing life-transforming medicines while tackling some of the most pressing scientific challenges today.

Watch the videos

Gene Therapy with
Targeted Delivery

The potential benefits of today’s gene therapies are limited by high dose requirements and systemic delivery methods, which often lead to increased risks of toxicity and off-target transduction. To combat these limitations, we are pioneering the development of next generation gene therapies that are delivered intrathecally to target the heart and skeletal muscle. This targeted mode of delivery may offer significant dose reduction, with the goal of an enhanced safety profile while remaining efficacious.

Learn more about our approach to gene therapy

2022_insmed_nh110958_2-square

RNA End-Joining Technology

Our RNA end-joining (REJ) technology may offer a solution for a second limitation of today’s gene therapies – the inability to target large genes with current delivery methods. While adeno-associated virus (AAV) vectors are the preferred delivery method for gene therapies, they can only hold genes of a certain size. Our proprietary REJ platform enables us to split large genes into multiple AAV capsids and reconstitute them once administered. This technology has the potential to unlock new possibilities for employing gene therapy to treat diseases caused by large genes that have historically not been viable targets for gene therapy.

Protein Deimmunization

Immunogenicity presents challenges for both gene therapies and protein therapeutics. For gene therapies, it prevents patients from receiving repeat doses and can limit the use of a vector to which patients may have developed natural immunity. For protein therapeutics, it limits the usefulness of current therapies and stifles innovation in the development of new ones. Our proprietary protein deimmunization platform, Deimmunized by Design® (DbD), offers a potential solution by using artificial intelligence to reengineer proteins to silence their immunogenic properties while maintaining their therapeutic activity. Using the DbD platform, we are working to develop re-dosable gene therapies to potentially unlock new, high unmet need disease targets as well as protein therapeutics with the goal of being less immunogenic.

Learn more about our proprietary protein deimmunization platform

2022_Insmed-1257_2-1

Proprietary Manufacturing

Our new, proprietary protein manufacturing platform has the potential to reduce the cost, time, and complexity of producing therapeutic proteins and viral vectors for gene therapy. We are advancing a ground-breaking technique that addresses the complexity and cost of current cell production approaches by taking advantage of the unique properties of algae. With potential benefits including rapid scalability, no plasmid transfection, low production costs, and ease of culturing and maintenance, we believe this platform may fundamentally transform protein manufacturing.

Synthetic Rescue

Despite ongoing advances in genetic technologies, the successful treatment of many genetic diseases remains out of reach. Our leading synthetic rescue platform provides a possible solution to this challenge by identifying targets outside the disrupted biological pathway which, when modulated, restore cell health. This scalable platform couples cutting-edge molecular and cell biology techniques with sophisticated analysis of human genetics datasets. Following robust validation of new targets, we select the best drug modality (e.g., small molecule, oligonucleotide, or gene therapy) to potentially deliver powerful new ways of treating a broad range of serious and rare diseases.

2022_insmed_nh110900_2

The platforms and technologies that comprise our early-stage research pillar are uniquely synergistic with one another and complementary to the rest of our business.

Taken together, they have the potential to change how we think about treating rare diseases and deliver real and meaningful results to patients. As we advance our fourth pillar, we also continue to assess new and attractive early-stage platform technologies that will complement our existing programs and bring us closer to achieving our mission.

Learn about our Other pillars

ARIKAYCE® (amikacin liposome inhalation suspension)

Learn more

Brensocatib

Learn more

Treprostinil Palmitil Inhalation Powder (TPIP)

Learn more

Footer

Insmed
  • Our Culture
    • Mission & Vision
    • Careers
  • Our Patients
    • Disease Areas
    • Resources
    • Patient Access
  • Our Science
    • Our Pillars
      • ARIKAYCE®
      • Brensocatib
      • TPIP
      • Early-Stage Research
    • Pipeline
    • Clinical Trials
    • Investigator-Initiated Research
    • Grants & Funding
  • Our Responsibility
    • Our Commitments
    • Inclusion at Insmed
    • Global Healthcare Compliance
    • Policies and Disclosures
  • About
    • Management Team
    • Board of Directors
    • Partnerships
  • Investors
  • News Releases
  • Events & Presentations
  • Earnings Results
  • FIlings
    • Annual Reports
    • SEC Filings
  • Corporate Governance
  • Responsibility Reports
  • Stock Information
  • Investor FAQs
  • Analyst Coverage
  • Email Alerts
LinkedInYouTube

Contact Us

Job Opportunities

© 2024 Insmed Incorporated. All Rights Reserved.

Terms of Use Privacy Policy California Notice at Collection U.S. Consumer Health Data Privacy Policy Your Privacy Choices

Culture
Patients
Science
logo
  • Culture
    • Mission & Vision
    • Careers
  • Patients
    • Disease Areas
    • Resources
    • Patient Access
  • Science
    • Our Pillars
      • ARIKAYCE®
      • Brensocatib
      • TPIP
      • Early-Stage Research
    • Pipeline
    • Clinical Trials
    • Investigator-Initiated Research
    • Grants & Funding
  • Responsibility
    • Our Commitments
    • Inclusion at Insmed
    • Global Healthcare Compliance
    • Policies and Disclosures
  • About
    • Management Team
    • Board of Directors
    • Partnerships
  • Investors
    • News Releases
    • Events & Presentations
    • Earnings Results
    • Filings
      • Annual Reports
      • SEC Filiings
    • Corporate Governance
    • Responsibility Reports
    • Stock Information
    • Investor FAQs
    • Analyst Coverage
    • Email Alerts

LinkedIn YouTube

Contact Us Job Opportunities

© 2024 Insmed Incorporated.
All Rights Reserved.

Terms of Use Privacy Policy U.S. Consumer Health Data Privacy Policy