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Experience across the entire process of gene therapy development

Leadership from the lab, to trials and through successful production Contact us

Why talk to Precision about your gene therapy project? Perhaps because we are purpose-built to solve the complex challenges posed by precision medicines like gene therapies. We’ve partnered with leading biotech and pharma companies to develop FDA and EMA approved treatments. Our services span from comprehensive biomarker assay suites to CRO services to leading the build and expansion of over 60 gene and cell therapy facilities. 

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Purpose-built to deliver on the promise of gene therapy

Gene therapy development has a wide range of challenges that include a dynamic regulatory environment, pre-existing and treatment-induced immunity to the viral vectors, intricate manufacturing processes, locating and recruiting patients.

Precision’s transformational approach to preclinical development, clinical trials and manufacturing ensures that your project has the comprehensive services and expertise needed to create solutions for every gene therapy development challenge, from IND-enabling assays to commercialization.

10 cell, gene and novel therapies industrialized
12 sample processing locations worldwide
50 rare disease clinical trials and trials for 100+ orphan projects
350 IVD and CDx global regulatory submissions and registrations

Solving complex challenges in gene therapy development

Proven expertise in gene therapy along with integrated development capabilities helps compress timelines and find efficiencies in every stage. Precision accelerates gene therapy development from the late pre-clinical phase through commercialization.

  • Explore

    Specialty Lab Services

    • NAb and TAb assays 
    • Immunogenicity testing e.g. ELISpot and Fluorospot
    • Viral capsid and transgene expression assays
    • Distribution and persistence assays
    • Central laboratory testing services for global trials
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    manufacturing

    Manufacturing Solutions

    • End-to-end services for the entire manufacturing life cycle
      • Planning and designing
      • Building and testing
      • Deploying and operating
      • Scalability and flexibility
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    clinical-laboratory-support

    Clinical Trial Design

    • Full service CRO for global trials
    • Patient recruitment and enrollment: specialization in rare disease
    • Expertise with current regulatory environment
    • Operational excellence in cell therapy study conduct
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    regulatory-solutions

    Regulatory Consulting

    • Well versed in EMA and FDA regulatory requirements 
    • Access to FDA officials to review proposed approaches
    • Able to advise on the latest safety, efficacy and quality standards
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    sample-processing

    PBMC Processing Services & Clinical Sample Processing

    • Clinical sample kit development
    • PBMC processing at 11 labs in five continents
    • Global sample logistics, shipping and biostorage
    • Real-time sample data analytics
    • virtual Sample Inventory Management (vSIM)
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    commercialization

    CDx / LDT Development

    • Co-development timelines of treatment and CDx
    • TAb and NAb companion diagnostic development
    • Extensive experience with multiple AAV serotypes
    • Access, reimbursement, and channel analysis and strategy

     

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Delivering evidence at every critical inflection point

With leadership in immunogenicity, a regulatory team that coordinates therapeutic and companion diagnostic strategy, and a focus on executing end-to-end clinical trials in oncology and rare disease, Precision is guiding successful gene therapy development for life sciences innovators.

  • IND-Enabling

    IND-Enabling

    Safety :

    • AAV Tab/NAb assays primates
    • PK transgene assays
    • ELISpot - T cell response assays
    • Tissue Distribution assays

  • Clinical Proof

    Clinical Proof

    Safety :

    • TAb/NAb assays

    Efficacity: 

    • Comprehensive PK/PD assays
    • DFA Diagnostic presubmission
    • Protocol design
    • End-to-end trial execution
    • Branded recruitment campaigns


    Value :

    • Burden of illness studies
    • Indirect treatment comparisons
    • Scientist platform

  • Pivotal

    Pivotal

    Safety :

    • NAb assays - patient recruitment
    • Module PMA submissions
    • Complete LDT analytical studies
    • Diagnostic market access strategy

    Efficacity: 

    • Pivotal Trial Execution

    Value :

    • Endpoint design
    • Pricing and access strategy
    • Brand positioning 

  • Commercial

    Commercial

    Safety :

    • NAb companion diagnostic

    Efficacity: 

    • Postmarket trial

    Value :

    • Brand marketing
    • Budget impact
shot of sterile pharmaceutical manufacturing laboratory

Industrializing novel and complex therapies

Precision has vast experience in designing and building GMP-compliant cell and gene therapy facilities, using cutting-edge modular, emerging bioreactor, and single-use technologies. Our manufacturing playbook—which has been used by the leading cell and gene therapy organizations—has led to the development of the largest and most mature ATMP manufacturing footprint in the world.

6

in tech ops capital investments being managed

Optimizing Successful Gene Therapy Development

Optimizing successful development of viral vector gene therapies, gene therapy trials and companion diagnostics

Download The White Paper

Top leadership ratings for a CRO in the ISR Report 2024

  • CRO Awards Leadership2024Compatability
  • CRO Awards Leadership2024Capabilities
  • CRO Awards Leadership2024Reliability
  • CRO Awards Leadership2024Quality
  • CRO Awards Leadership2024Expertise

Our latest insights on gene therapy development

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Read: Gene Therapy Assays: Regulatory Compliance with Unified Immunogenicity Gene Therapy Assays: Regulatory Compliance with Unified Immunogenicity

Translational Research - Regulatory - Assays - Gene Therapies

Gene Therapy Assays: Regulatory Compliance with Unified Immunogenicity

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  • Deborah P. avatar

    Deborah P.
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    Maham A.
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Read: Therapeutic Genome Editing – Reshaping the Landscape of Advanced Therapies Therapeutic Genome Editing

Regulatory - Gene Therapies

Therapeutic Genome Editing – Reshaping the Landscape of Advanced Therapies

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    has not third author: true, (SizeLimitingPyMap: {main={hs_id=169567234066, hs_child_table_id=0, hs_updated_at=1723217613021, hs_published_at=1739807653319, description=Precision Experts are among the most experienced specialists in clinical research. We focus our hiring strategy on select indications to deliver excellence across each role. , avatar=Image{width=800,height=800,url='https://5014803.fs1.hubspotusercontent-na1.net/hubfs/5014803/Precision%20Expert.png',altText='Precision Expert',fileId=164175466823}, lastname=Experts, hs_initial_published_at=1718988967067, hs_created_by_user_id=26433386, hs_created_at=1717603871601, hs_is_edited=false, hs_deleted_at=0, name=Precision, slug=precision-experts, hs_updated_by_user_id=51739740}, second={}, third={}})
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    Precision Experts
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Read: Navigating Cell and Gene Therapy Acquisitions: A Strategic Blueprint Navigating Cell and Gene Therapy Acquisitions: A Strategic Blueprint

Manufacturing - Cell Therapies - Gene Therapies

Navigating Cell and Gene Therapy Acquisitions: A Strategic Blueprint

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    has not third author: true, (SizeLimitingPyMap: {main={hs_id=164426546550, hs_child_table_id=0, hs_updated_at=1716454235474, hs_published_at=1739807653319, description=Groundbreaking biotech entrepreneur, executive, and investor, dedicated to delivering transformative treatments to the patients who need them. Pioneered the industrialization of advanced therapeutics, including some of the earliest commercially approved cell and gene therapies, radioligand treatments, RNA-based therapies, and cutting-edge biologics. Created the leading global biologics and advanced therapy engineering consulting firm and developed the largest cell and gene therapy manufacturing footprint in the world. Deeply committed to advancing next-generation medicines, serves on multiple boards, philanthropic organizations, and patient-advocacy groups, including IQHQ, Alliance for Regenerative Medicines, and Alliance for mRNA Medicines., avatar=Image{width=720,height=812,url='https://5014803.fs1.hubspotusercontent-na1.net/hubfs/5014803/PfM%20Website/Headshots/Headshots%20no%20background/Anshul-Mangal.png',altText='Anshul-Mangal',fileId=167370347684}, linkedin=https://www.linkedin.com/in/amangal/, team=[{id=163072475007, name='Precision for Medicine Leadership'}], lastname=Mangal, hs_initial_published_at=1713174153602, hs_created_by_user_id=62155952, hs_created_at=1713169484118, hs_is_edited=false, hs_deleted_at=0, name=Anshul, job=President, Manufacturing Solutions, slug=anshul-mangal, hs_updated_by_user_id=9297597}, second={hs_id=164426546553, hs_child_table_id=0, hs_updated_at=1716454239819, hs_published_at=1739807653319, description=Life sciences leader who helped pioneer the industrialization of biologics and advanced therapies. Deep expertise in small and large molecules, honed through work with a range of life science organizations, from start-ups and academic centers to financial institutions and CMOs/CROs. Spearheaded significant global turnkey facility builds, automation, validation, engineering, and serialization track and trace, deploying billions in capital across the life sciences sector. Led the creation of the Advanced Therapy Manufacturing Playbook which has helped develop the world’s largest advanced therapy manufacturing footprint. Actively serves on multiple boards including academic, philanthropic, biotech, and financial investment institutions., avatar=Image{width=576,height=576,url='https://5014803.fs1.hubspotusercontent-na1.net/hubfs/5014803/PfM%20Website/Headshots/Headshots%20no%20background/Tony%20Khoury.webp',altText='Tony Khoury',fileId=165893018152}, linkedin=https://www.linkedin.com/in/tony-khoury-7470755/, team=[{id=163072475007, name='Precision for Medicine Leadership'}], lastname=Khoury, hs_initial_published_at=1713174153602, hs_created_by_user_id=62155952, hs_created_at=1713169549303, hs_is_edited=false, hs_deleted_at=0, name=Tony, job=Executive Vice President, Manufacturing Solutions, slug=tony-khoury, hs_updated_by_user_id=9297597}, third={}})
  • Anshul M. avatar

    Anshul M.
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    Tony K.
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