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Cell and Gene Therapy (CGT)

Published: April 2025
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  1. Introduction:

Cell and Gene Therapy (CGT) is an advanced area of medicine that uses genetic modification and cellular engineering to treat or cure diseases, particularly those with limited treatment options. It includes two main approaches:

  • Cell Therapy – The use of living cells to repair, replace, or regenerate damaged tissues or organs. Examples include:
    • CAR-T cell therapy (e.g., Kymriah, Yescarta) for cancer treatment
    • Stem cell therapies for regenerative medicine
  • Gene Therapy – The modification of genetic material to treat or prevent disease by inserting, deleting, or correcting faulty genes. Examples include:
    • Luxturna for inherited retinal disease
    • Zolgensma for spinal muscular atrophy

Certain treatments fall under both cell and gene therapy categories. These therapies function by modifying genes within specific cell types before introducing them into the body.

This combined approach addresses some limitations of using gene or cell therapy individually. Utilizing a patient’s own cells eliminates the need for a donor match, and modifying cells in a laboratory setting is often more manageable than making genetic changes directly within the body.

 

2. Key Trends & Advancements in CGT

​The Cell and Gene Therapy (CGT) sector is experiencing rapid advancements, marked by significant trends that are reshaping the landscape of medical treatment. Key developments include:​

  • Expansion of Approved Therapies
  • Increased FDA Approvals: As of March 2024, the FDA has approved 36 gene therapies, with an additional 500 therapies in the pipeline and expectations of 10–20 approvals annually by 2025.
  • Diversification Beyond Oncology: There is a notable shift toward non-oncology indications, with 51% of newly initiated gene therapy trials focusing on areas such as rare genetic disorders and neurodegenerative diseases, up from 39% in the previous year. ​
  • Advancements in Gene Editing Technologies
  • CRISPR-Cas9 Innovations: The development of next-generation CRISPR techniques enables precise correction of genetic mutations without introducing double-stranded DNA breaks, enhancing safety and efficiency in gene editing.
  • Base and Prime Editing: These refined gene editing methods allow for the accurate "find and replace" of specific DNA sequences, offering potential treatments for a wide array of genetic diseases.
  • Automation and Manufacturing Innovations
  • Automated Production Systems: Companies like Cellares are developing robotic systems, such as the Cell Shuttle, to automate cell therapy manufacturing, aiming to reduce costs and increase the accessibility of these therapies. ​
  • Closed-System Manufacturing: The adoption of closed-system manufacturing enhances product safety and scalability, addressing challenges in producing complex therapies. ​
  • Integration of Artificial Intelligence (AI)
  • Enhanced Data Analytics: AI is being utilized to analyze complex biological data, aiding in the identification of therapeutic targets and the design of personalized treatment strategies. ​
  • Predictive Modeling: AI-driven models are improving the prediction of patient responses to therapies, optimizing clinical trial designs, and accelerating the development of effective treatments. ​
  • Emergence of Personalized Medicine
  • Tailored Treatments: Advances in genomic sequencing are facilitating the development of therapies customized to individual genetic profiles, enhancing efficacy and reducing adverse effects.
  • Regulatory Support: Regulatory agencies are increasingly approving personalized medications, with the FDA approving 26 personalized medicines in 2023, including six cell and gene-based therapies. ​
  • Addressing Manufacturing Challenges
  • Scalability Solutions: Innovations in manufacturing processes, such as automation and closed systems, are being implemented to overcome scalability issues and meet the growing demand for CGTs.
  • Cost Reduction Efforts: Strategies are being developed to reduce production costs, making therapies more affordable and accessible to a broader patient population. ​
CGT - Key Trends & Advancements

3. Approved CGTs - Sales & Forecast

Several Cell and Gene Therapies (CGTs) have received regulatory approval from agencies like the FDA (U.S.), EMA (Europe), and other global regulators.

TherapyCompanyIndicationTypeMechanism
Zolgensma (onasemnogene abeparvovec-xioi)NovartisSpinal Muscular Atrophy (SMA)Gene TherapyAAV-based gene replacement
Luxturna (voretigene neparvovec)Spark Therapeutics (Roche)Inherited retinal disease (RPE65 mutation)Gene TherapyAAV-based gene therapy
Roctavian (valoctocogene roxaparvovec)BioMarinHemophilia AGene TherapyAAV-based gene therapy for factor VIII production
Skysona (elivaldogene autotemcel, eli-cel)Bluebird BioCerebral Adrenoleukodystrophy (CALD)Gene TherapyLentiviral vector-based gene correction
Lyfgenia (lovotibeglogene autotemcel, lovo-cel)Bluebird BioSickle Cell DiseaseGene TherapyLentiviral vector-based gene therapy
Hemgenix (etranacogene dezaparvovec)CSL BehringHemophilia BGene TherapyAAV-based factor IX gene therapy
Alofisel (darvadstrocel)TakedaPerianal fistulas in Crohn’s DiseaseCell TherapyAllogeneic stem cell therapy
StrimvelisOrchard TherapeuticsADA-SCID (Bubble Boy Syndrome)Cell TherapyEx vivo stem cell gene therapy
Kymriah (tisagenlecleucel)NovartisB-cell Acute Lymphoblastic Leukemia (ALL), LymphomaCAR-T TherapyAutologous CAR-T targeting CD19
Yescarta (axicabtagene ciloleucel)Gilead (Kite Pharma)Large B-cell Lymphoma (LBCL), Follicular LymphomaCAR-T TherapyAutologous CAR-T targeting CD19
Tecartus (brexucabtagene autoleucel)Gilead (Kite Pharma)Mantle Cell Lymphoma (MCL)CAR-T TherapyAutologous CAR-T targeting CD19
Breyanzi (lisocabtagene maraleucel)Bristol Myers SquibbLarge B-cell Lymphoma (LBCL)CAR-T TherapyAutologous CAR-T targeting CD19
Abecma (idecabtagene vicleucel)BMS & 2seventy bioMultiple MyelomaCAR-T TherapyAutologous CAR-T targeting BCMA
Carvykti (ciltacabtagene autoleucel)Janssen & BiotechMultiple MyelomaCAR-T TherapyAutologous CAR-T targeting BCMA
CGT - FDA Approved Cell & Gene Therapies

 

4. Pipeline Analysis and Expected Approval Timelines

​The cell and gene therapy (CGT) sector is poised for significant growth, with numerous therapies in the pipeline and several anticipated approvals on the horizon.

Below is an analysis of the current pipeline and expected approval timelines:​

CGT - Key Emerging Therapies - Current timelines
CGT - Key Emerging Therapies - Expected Approvals

5. Market Size & Forecasting

The global cell and gene therapy market was valued at ~$13.90 billion in 2024 and is anticipated to be valued at ~$105.83 billion by 2033, registering a CAGR of 20-22% over the forecast period

Global CGT Market - Overview


 

Unmet Needs

The cell and gene therapy (CGT) sector has seen tremendous advancements, but several critical unmet needs remain:

CategoryUnmet NeedChallenges
Manufacturing & ScalabilityHigh production costsComplex processes make CGTs expensive
Limited production capacityInsufficient facilities for large-scale production
Need for automationManual processes increase variability and cost
Delivery & TargetingIn vivo gene delivery issuesEfficient, safe delivery mechanisms are needed
Immune response risksImmune reactions to viral vectors limit efficacy
Precise gene editing neededOff-target effects can cause unintended mutations
Affordability & ReimbursementHigh therapy costsCGTs can cost $1M–$3M per patient
Limited insurance coverageHealth systems struggle with one-time curative payments
Need for innovative payment modelsOutcome-based or installment-based payments required
Patient Access & LogisticsLong manufacturing timesSome cell therapies take weeks to produce
Limited treatment centersOnly specialized hospitals can administer CGTs
Cold chain challengesMany CGTs require cryopreservation, complicating transport
Efficacy & DurabilityUncertain long-term durabilitySome therapies may lose effectiveness over time
Limited indicationsMost CGTs target rare diseases, broader applications needed
Resistance mechanismsSome cancers develop resistance to CAR-T therapies
Regulatory & Ethical ChallengesFast-evolving fieldRegulations struggle to keep pace with advancements
Ethical concernsGermline editing raises bioethical issues
Data gapsNeed for more real-world evidence on safety & efficacy



 

6. Competitive Landscape and Market Positioning

​The Cell and Gene Therapy (CGT) sector is experiencing rapid growth, characterized by significant investments, strategic partnerships, and a dynamic competitive landscape.​

CategoryCompany/OrganizationMarket Strategy & Positioning
Big Pharma LeadersNovartis, Pfizer, AstraZenecaInvesting in acquisitions and partnerships to expand CGT pipelines
Biotech InnovatorsBluebird Bio, CRISPR TherapeuticsFocus on pioneering gene-editing therapies but facing financial constraints
Contract Manufacturing (CDMOs)Catalent, Lonza, WuXi AppTecSupporting large-scale CGT production for biotech and pharma firms
Emerging PlayersGracell Biotechnologies, Century TherapeuticsDeveloping next-generation allogeneic and off-the-shelf therapies



 

7. Target Opportunity Profile (TOP) & Benchmarking

The Target Opportunity Profile (TOP) and Benchmarking framework help assess market potential, competitive positioning, and key differentiators in the CGT landscape.

  • Target Opportunity Profile (TOP)
CategoryDetails
Target IndicationsRare genetic disorders, hematologic cancers, autoimmune diseases
Market Size~$13.9B (2024), expected to grow to ~$105.83B by 2033
Unmet NeedsHigh treatment costs, scalability challenges, long-term durability concerns
Growth DriversCRISPR advancements, regulatory approvals, investment in manufacturing
Key StakeholdersBig pharma, biotech firms, CDMOs, regulatory agencies, payers



 



 

  • Competitive Benchmarking
ParameterNovartis (Kymriah, Zolgensma)Pfizer (Gene Therapy Portfolio)Bluebird Bio (LentiGlobin, Zynteglo)
Therapy TypeCAR-T (Kymriah), Gene therapy (Zolgensma)Multiple gene therapies in developmentLenti-based gene therapies for blood disorders
Key IndicationsLeukemia, Spinal Muscular Atrophy (SMA)Rare genetic disorders, oncologySickle cell disease, β-thalassemia
Regulatory ApprovalsFDA, EMA (Multiple approvals)In development (Limited commercial approvals)FDA-approved but facing commercialization hurdles
Revenue (2024)~$2.5B (Zolgensma, Kymriah)~$1B expected from gene therapy segment<$500M (struggling with adoption)
Manufacturing ModelIn-house + CDMO supportIn-house R&D with partnershipsOutsourced manufacturing
ChallengesHigh costs, reimbursement hurdlesDevelopment delays, scalabilityFinancial struggles, slow market adoption



 


 

 

 

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