© CIDETEC 2017

Iraida Loinaz Nanomedine Business Manager

GMP Pilot Plant

Facilities and capacities 19/09/2017

2 C O N F I D E N T I A L

The production of innovative nanopharmaceuticals in quantity and quality (GMP)

required for SMEs to enter clinical trials remains a challenge:

•! Medium size companies with limited R&D facilities, use their existing manufacturing plants, which are too big and too busy for R&D purposes.

•! Small companies lack of resources to up-scale and implement GMP manufacturing.

The Problem

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3 C O N F I D E N T I A L

NanoPilot is a four-year long project which objective is to set-up a pilot plant operating under GMP (Good Manufacturing Practices) for the production of polymer-based nanopharmaceuticals.

Pilot plant’s ambition is to accelerate the development of nanomedicine, currently in its infancy within the pharmaceutical sector.

4 research groups, 3 SMEs and 2 industries, all of them with significant track record in knowledge creation and innovation in their respective domains of expertise, have joined forces in NanoPilot to guarantee the successful outcome of the proposed project

NanoPilot

Funded under European Union Framework Programme for Research and Innovation Horizon 2020

Project acronym: NanoPilot Grant Agreement no: 646142 Start Date: January 1st, 2015 End Date: December 31st, 2018 Project Budget: 6.28 M Euro Type: Research and Innovation Action

The research from the NanoPilot project leading to these results has receiving funding from the EU Framework Programme for Research and Innovation HORIZON 2020 under Grant Agreement 646142.

4 C O N F I D E N T I A L

Objectives NanoPilot will set-up a flexible and adaptable pilot plant operating under GMP for the production of small batches of polymer based nanopharmaceuticals, which exhibit significant potential in the field of drug-delivery particularly for the design of second generation nanopharmaceuticals.

5 C O N F I D E N T I A L

Objetives based on industrial needs

Flexibility and Adaptability

A design which will look for

a compromise between:

specialization, flexibility and

economic viability.

Production of small GMP batches for clinical trials

The pilot plant will provide the

small quantities of products that

technology developers will

require for full preclinical and

first clinical validation

The pilot plant will provide the

small quantities of products that

The big pharma will require

technology which is robust and

translatable, that has completed

full toxicological preclinical phase

and had been validated in first

clinical trials.!

6 C O N F I D E N T I A L

Dedicated to technology transfer from lab to pilot scale – Unlike CMOs which are interested in the production of big batches.

Cost-effective: Includes small equipment to produce only required amount of doses.

Flexible to the customer needs: Campaign production will allow the implementation of different processes in the same facilities.

Cost-effectiveCost-effectiverequiredrequired amount

The Pilot Plant

7 C O N F I D E N T I A L

•! Understand the process by DoE (process characterization).

•! Development and scaling. •! QC strategy planning. •! QC processes development. •! Verification and validation. •! Sterility – Validation.

Technology transfer / Design of Manufacturing process

Launching Stage& Excution stage (I)& Excution stage (II) – GMP&

•! Data Collection and Gap analysis.

•! Current lab process evaluation.

•! Starting materials (providers).

•! Manufacturing. •! Packaging. •! ! •! !

8 C O N F I D E N T I A L

Polymer Nanosystem Characterisation: difficulties

A4F-MALS-RI SEC-RI SEC-triple detector

NanoZS Malvern

Vasco 2 Cordouan Technologies

90 Plus Brookhaven

9 C O N F I D E N T I A L

Polymer Nanosystem Characterisation: difficulties

DLS Malvern Cordouan Technologies

Brookhaven

Model NanoZS Vasco 2 90 plus

Observation angle 173º 135º 90º

10 C O N F I D E N T I A L

Continuous flow reactors

Cost-effective production

Batch production

11 C O N F I D E N T I A L

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Production

QC

Flexible to customer needs

12 C O N F I D E N T I A L

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QC Area

Storaging Area Packaging

Production Area

13 C O N F I D E N T I A L

Hyaluronan based particles

Peptidic antigens A short interfering RNA (siRNA)

nanoformulation

Production of three different

nanopharmaceuticals A treatment for

interstitial cystitis/painful bladder

syndrome (IC/PBS).

A HIV nanovaccine formulated for

intranasal vaccination

Topical treatment of ocular pain associated with dry eye syndrome

(DES).

14 C O N F I D E N T I A L

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© CIDETEC 2017