Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3...

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Cell and Gene Therapy Supply Chain Challenge or Opportunity? December 2016

Transcript of Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3...

Page 1: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Cell and Gene Therapy Supply ChainChallenge or Opportunity?

December 2016

Page 2: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Agenda

• Introduction to the Cell and Gene Therapy Catapult

• What are cell and gene therapies?

• Clinical impact

• Supply chain terminology

• Auto, allo, vector

• Industry growth/potential

• Challenges - current supply chain

• Opportunities – future supply chain

Introduction 2

Page 3: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Why cell and gene therapy

Introduction 3

An opportunity to build a large-scale industry delivering health and wealth to the UK.

Identified significant and growing unmet healthcare needs that cell and gene therapy could address

The UK is at the leading-edge of the cell and gene therapy industry.

01 0302

Page 4: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Introduction to the Cell and Gene Therapy Catapult

Page 5: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

The Catapults

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• Part of a world-leading network of technology and innovation centres

• Bridge the gap between businesses, academia, research and government

• Access technical facilities and expertise to help adopt, develop and exploit innovations

• Established by Innovate UK

Growing the UK cell and gene therapy industry, deliveringhealth and wealth.

“ “

Page 6: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Everything you need under one roof

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Our capabilities complement each other across the cell and gene therapy lifecycle

Safe and effective

Scalable

Affordable

Non-clinical safety

Clinical operations

Regulatory expertise Industrialisation˃ ˃ ˃

Investment in infrastructure -GMP facility and state-of-the-art laboratories

Regulatory expertise Industrialisation˃ ˃

Health economics and market access Industrialisation˃

Page 7: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

UK industry growth 2012 to 2015

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£400m+Investment 2015Investment attracted by UK companies in 2015 vs. £35m in 2012¹

+1,000Jobs created are up from 540 in 2012 (expected to exceed 2,000 in 5yrs)

42ATMP therapy developers50% are rapidly growing

Growth in UK clinical trials since 2013

Growth in the number of ATMP therapy companies since 2012

+50%GMP footprint 22 facilities/11,800m². A 50% increase vs. 2013

+50%

£400m+

90%

¹Excludes Smith & Nephew acquisition of Healthpoint for $782M in 2012

Page 8: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Cell and gene therapies

Page 9: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Potential of cell therapies

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Potential cell sources include:

• Pluripotent stem cells

• Adult stem cells e.g. MSCs

• Hematopoietic stem cells

• Immune cells

Stroke Traumatic brain injuryAlzheimer’s diseaseParkinson’s disease

Missing teeth

Wound healing

Bone marrowtransplantation(currently established)

Spinalcord injury

OsteoarthritisRheumatoidarthritis

Baldness

Blindness

Deafness

Lou Gehrig’sdisease

Myoardialinfarction

Musculardystrophy

Diabetes

Multiple sites:cancers

Crohn’sdisease

Page 10: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Major milestones and key data events

Introduction 10

Page 11: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Example – T cell therapy

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Collect patient’s white blood cells

Isolate and activate T cells

Grow and expand number of T cells

Infuse patient with engineered T cells

Engineer T cells with CAR or TCR gene

Modified from Kite Pharma

Page 12: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Terminology and logistics challenge

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Courtesy of Lonza

Allogeneic Autologous

Page 13: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Challenges in current supply chain

Page 14: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Low volume - orphan indication (20 patients/yr)

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Manufacture Patient

NB

• One EU manufacturing site supplying EU, one US site supplying North America.

• Contingency planning is vital.

• No consolidation with other shipments possible.

• Maximum visibility at all points during shipping.

Bone marrow donation

Volume • 20/yr

Temp • Controlled ambient

Container • Blood bag

Shelf Life • <24hrs

Viral vector

Volume • “Large” single shipment

Temp • -80C

Container • Vials

Shelf Life • Months

Final product

Volume • 20/yr

Temp • 2-4C

Container • 50-80ml in 120ml syringe

• Shipped in metal box

Shelf Life • < 24 hrs

Page 15: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

High volume allo therapy (100,0000 patients/yr (globally)

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Manufacture

Patient

NB

• Large central manufacturing

• Deliveries will be restocking as opposed to patient specific

• Consolidation of final product is essential

• Different product formulations and final packaging may decrease number of doses

per shipper significantly.

• Significant space required for dry shippers, insulated boxes, dry ice and LN2

storage

Initial Donation

This will only required

once as will form “bank”

for later therapies

Final Product

Volume • 100s / hospital

Temp • -180C

Container • Dry shipper (13l LN2=27kg)

• 500 vials/shipper

Shelf Life • 10 dys

Satellite

Satellite

Satellite Patient

Patient

Page 16: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Growth = challenge

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Page 17: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Hidden challenges

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Cell is the product Manual supply chain

Pack/label Goods in/out Admin

Complicated systems Variable supply chain

Du

rati

on

European centre

Page 18: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Benchmarking

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• Limited number of specialist providers

• White glove service

• “No” shipment integration

• Data from clinical trial is critical

• Cost “irrelevant”

• Limited development of shipping technologies

Page 19: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Opportunities in future supply chain

Page 20: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Updating the supply chain

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Digital real time tracking

Distribution hub

Improved container

Thaw in clinic

Page 21: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

This is not a new challenge

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Validated, track and trace Cold Supply Chain for “cells”

High volume, high value shipping 1.85m cell therapies per year

t

Page 22: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Opportunities – working together to…..

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…repurpose existing technology. …enable a global industry.

…meet patient/clinician needs. …create a viable, supply chain.

Page 23: Cell and Gene Therapy Supply Chain Challenge or Opportunity? · Why cell and gene therapy 3 Introduction An opportunity to build a large-scale industry delivering health and wealth

Cell and Gene Therapy Catapult

12th Floor Tower WingGuy’s HospitalGreat Maze PondLondon SE1 9RT

+44 (0)20 3728 [email protected]: @CTCatapult

Introduction 23


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