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Explore end-to-end solutions throughout development — from portfolio optimization and regulatory strategy, to Phase I-IV clinical trials, market access planning, and more.

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Overview

Portfolio management and asset valuation
- Building patient insights into assets, profile and claims
- Portfolio optimization
- Asset valuation and indication prioritization
- Early evidence review
- Integrated development strategy and planning
Early development and innovation
- Early Phase Clinical Trials
- Site Alliance Network and KOL Engagement
- Quantitative Clinical Pharmacology and Model-Informed Drug Development (MIDD)
- Regulatory Strategy
- Real World Evidence
- Market Access Consulting, Strategy, and Delivery
- Dedicated early phase clinical units
- Biomarker and Genomic Medicine Strategy
- Integrated evidence generation planning
- Clinical Logistics
- Medical Communications
Integrated clinical development
- Phase IIB-IV Clinical Trial Solutions
- Real World Evidence
- Quantitative Clinical Pharmacology and Model-Informed Drug Development (MIDD)
- Protocol-Driven, Customized Site Solution Strategy
- Patient Inclusion
- Regulatory Strategy, Submissions, Compliance, and Outsourcing
- Market Access Strategy and Delivery
- Clinical Logistics
- Clinical Development Technology Optimization
- Integrated evidence generation planning
- Medical Communications
- Drug Safety and Pharmacovigilance
Approval and access
- Global Regulatory Submissions and Outsourcing
- Real World Evidence
- Compliance and Risk Management
- Market Access Strategy and Delivery
- Integrated evidence generation planning
- Medical Communications
- Drug Safety and Pharmacovigilance
- Health economics modelling and simulation
Value substantiation lifecycle management
- Real-World Evidence, Market Access Strategy and Planning
- Regulatory Compliance
- Lifecycle Optimization
- Integrated evidence generation planning
- Medical Communications
FSP delivery models in clinical trials
- Gain an advantage through FSP
Operational excellence
Medical affairs for clinical trials
Clinical study start up
ParexelAI
- Parexel and Paradigm
- Leveraging AI in clinical development

Reliable, fast delivery

ParexelAI

ParexelAI is a proprietary AI-enabled clinical development suite that accelerates patient outcomes by combining Parexel's operational expertise with cutting-edge AI technology.

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Biotech
Therapeutic Expertise

Therapeutic Expertise

Utilize our expertise across therapeutic areas, combining innovative trial designs, leading clinical and regulatory expertise, global reach, and a passion for changing patient lives.

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THERAPEUTIC EXPERTISE

CROSS-THERAPEUTIC EXPERTISE

Overview

Cell and gene therapy
General medicine
- Gastroenterology CRO
- Respiratory CRO
- Endocrine & Metabolic CRO
- Cardiovascular CRO
- Women's health CRO
Infectious disease and vaccines
- Critical Care
- Hepatitis B
- Novel treatments in infectious disease
- Vaccines
Immunology
- Dermatology CRO
- Allergy CRO
Oncology CRO
- Bladder cancer CRO
- Breast cancer CRO
- Colorectal cancer CRO
- Gynecologic cancer CRO
- Head and neck cancer CRO
- Liver cancer CRO
- Lung cancer CRO
- Pancreatic cancer CRO
- Phase I oncology CRO
- Prostate cancer CRO
Neuroscience
- Neurology
- Psychiatry
Pediatrics
Rare Diseases
Hematology
- Non-malignant hematology
- Malignant hematology CRO
Ophthalmology CRO

RELATED INSIGHTS

Cell & Gene Unscripted: Real-world evidence

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About

We are one of the largest CROs in the world, speeding life-changing medicine to market by engaging patients With Heart™. Learn about who we are, what we do, and what we believe.

About Parexel

Overview

Leadership team
- Board of Directors
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- Ireland Gender Pay Data
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- Vulnerability Reporting Program
Meet us at an event
- Poster presentations at EHA 2026
- Parexel Biotech Incubator

Main Menu

Solutions

Explore end-to-end solutions throughout development — from portfolio optimization and regulatory strategy, to Phase I-IV clinical trials, market access planning, and more.

See solutions

WHAT WE DO

HOW WE DO IT

Overview

Portfolio management and asset valuation
- Building patient insights into assets, profile and claims
- Portfolio optimization
- Asset valuation and indication prioritization
- Early evidence review
- Integrated development strategy and planning
Early development and innovation
- Early Phase Clinical Trials
- Site Alliance Network and KOL Engagement
- Quantitative Clinical Pharmacology and Model-Informed Drug Development (MIDD)
- Regulatory Strategy
- Real World Evidence
- Market Access Consulting, Strategy, and Delivery
- Dedicated early phase clinical units
- Biomarker and Genomic Medicine Strategy
- Integrated evidence generation planning
- Clinical Logistics
- Medical Communications
Integrated clinical development
- Phase IIB-IV Clinical Trial Solutions
- Real World Evidence
- Quantitative Clinical Pharmacology and Model-Informed Drug Development (MIDD)
- Protocol-Driven, Customized Site Solution Strategy
- Patient Inclusion
- Regulatory Strategy, Submissions, Compliance, and Outsourcing
- Market Access Strategy and Delivery
- Clinical Logistics
- Clinical Development Technology Optimization
- Integrated evidence generation planning
- Medical Communications
- Drug Safety and Pharmacovigilance
Approval and access
- Global Regulatory Submissions and Outsourcing
- Real World Evidence
- Compliance and Risk Management
- Market Access Strategy and Delivery
- Integrated evidence generation planning
- Medical Communications
- Drug Safety and Pharmacovigilance
- Health economics modelling and simulation
Value substantiation lifecycle management
- Real-World Evidence, Market Access Strategy and Planning
- Regulatory Compliance
- Lifecycle Optimization
- Integrated evidence generation planning
- Medical Communications
FSP delivery models in clinical trials
- Gain an advantage through FSP
Operational excellence
Medical affairs for clinical trials
Clinical study start up
ParexelAI
- Parexel and Paradigm
- Leveraging AI in clinical development

Reliable, fast delivery

ParexelAI

ParexelAI is a proprietary AI-enabled clinical development suite that accelerates patient outcomes by combining Parexel's operational expertise with cutting-edge AI technology.

Learn more

Therapeutic Expertise

Utilize our expertise across therapeutic areas, combining innovative trial designs, leading clinical and regulatory expertise, global reach, and a passion for changing patient lives.

See expertise

THERAPEUTIC EXPERTISE

CROSS-THERAPEUTIC EXPERTISE

Overview

Cell and gene therapy
General medicine
- Gastroenterology CRO
- Respiratory CRO
- Endocrine & Metabolic CRO
- Cardiovascular CRO
- Women's health CRO
Infectious disease and vaccines
- Critical Care
- Hepatitis B
- Novel treatments in infectious disease
- Vaccines
Immunology
- Dermatology CRO
- Allergy CRO
Oncology CRO
- Bladder cancer CRO
- Breast cancer CRO
- Colorectal cancer CRO
- Gynecologic cancer CRO
- Head and neck cancer CRO
- Liver cancer CRO
- Lung cancer CRO
- Pancreatic cancer CRO
- Phase I oncology CRO
- Prostate cancer CRO
Neuroscience
- Neurology
- Psychiatry
Pediatrics
Rare Diseases
Hematology
- Non-malignant hematology
- Malignant hematology CRO
Ophthalmology CRO

RELATED INSIGHTS

Cell & Gene Unscripted: Real-world evidence

Learn more

Cell & Gene Unscripted: Rare and genetic diseases

Learn more

Insights

Our experts help you stay at the forefront of the industry — and ahead of change.

Learn more

NEW MEDICINES, NOVEL INSIGHTS

FEATURED INSIGHTS

PODCAST

Clinical Trialblazers

Step into the world of biotech leaders and hear the untold stories behind their journeys to bring breakthrough innovations to life.

Listen Now

Cell & Gene Unscripted

Honest conversations with the experts shaping what's next

Watch the series

Participate

Thinking about joining a clinical trial? Learn the drug development process, what it’s like to participate, how to find a trial, and answers to frequently asked questions.

Learn more

INTERESTED IN PARTICIPATING?

TRIAL SITES

Overview

Patient volunteers
- Advisory Council
Healthy volunteers
Patient advocacy groups
Patient stories

HEAR FROM REAL PATIENTS

Patient Stories

Learn more

About

We are one of the largest CROs in the world, speeding life-changing medicine to market by engaging patients With Heart™. Learn about who we are, what we do, and what we believe.

About Parexel

Overview

Leadership team
- Board of Directors
Global reach
Our Inclusion Strategy
Our sustainability strategy
Compliance, tax & privacy
- Compliance & Ethics
- Tax Strategy
- Privacy Policy
- Cookies and other web tracking
- Supplier Data Privacy Requirements
- Terms of Use
- French Gender Pay Data
- UK Gender Pay Data
- UK Modern Slavery Act Statement
- Ireland Gender Pay Data
- Supplier Inclusion Policy
- Vulnerability Reporting Program
Meet us at an event
- Poster presentations at EHA 2026
- Parexel Biotech Incubator

Cell & Gene Unscripted: Rare and genetic diseases

0 min

Published on: Sep 16, 2026

Rare diseases affect millions of people worldwide, yet many patients and families still face long journeys to diagnosis, limited treatment options, and significant barriers to accessing care. Advances in cell and gene therapy continue to accelerate, creating new possibilities for conditions that historically had few or no treatment options.

In this episode of Cell & Gene Unscripted, Gvantsa Kvantaliani, Associate Medical Director, Rare and Genetic Diseases, draws on her unique perspective as a physician, rare disease expert, patient advocate, and parent of a child with a rare genetic condition. She shares an honest and deeply personal look at the realities of living and working in the rare disease community with Parexel's Head of Cell & Gene Therapy and Early Phase, Chris Learn, Ph.D. M.B.A., P.M.P.

The conversation explores the challenges families face throughout the diagnostic and treatment journey, while highlighting the importance of early diagnosis, patient advocacy, and greater coordination across researchers, industry, healthcare systems and government to improve access to care.

Return to Insights Center

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Solutions

WHAT WE DO

HOW WE DO IT

Portfolio management and asset valuation

Early development and innovation

Integrated clinical development

Approval and access

Value substantiation lifecycle management

ParexelAI™

Reliable, fast delivery

Therapeutic Expertise

THERAPEUTIC EXPERTISE

CROSS-THERAPEUTIC EXPERTISE

General medicine

Infectious disease and vaccines

Immunology

Oncology

Neuroscience

Hematology

RELATED INSIGHTS

Insights

NEW MEDICINES, NOVEL INSIGHTS

FEATURED INSIGHTS

PODCAST

Cell & Gene Unscripted

Participate

INTERESTED IN PARTICIPATING?

TRIAL SITES

Patient volunteers

HEAR FROM REAL PATIENTS

About

Leadership team

Compliance, tax & privacy

Solutions

WHAT WE DO

HOW WE DO IT

Portfolio management and asset valuation

Early development and innovation

Integrated clinical development

Approval and access

Value substantiation lifecycle management

ParexelAI™

Reliable, fast delivery

Therapeutic Expertise

THERAPEUTIC EXPERTISE

CROSS-THERAPEUTIC EXPERTISE

General medicine

Infectious disease and vaccines

Immunology

Oncology

Neuroscience

Hematology

RELATED INSIGHTS

Insights

NEW MEDICINES, NOVEL INSIGHTS

FEATURED INSIGHTS

PODCAST

Cell & Gene Unscripted

Participate

INTERESTED IN PARTICIPATING?

TRIAL SITES

Patient volunteers

HEAR FROM REAL PATIENTS

About

Leadership team

Compliance, tax & privacy

Cell & Gene Unscripted: Rare and genetic diseases

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