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PowerPoint Presentation

PI: A/P Giorgia PASTORIN EL: Wei Jiang GOH TM: Shui ZOU Mentor: Dr Ju Wei WONG

EXOPLEXsTailoring Your Transfection Needs

EXOPLEXsAn Nanoparticle System

Drug cargoEnlarged ViewEXOPLEXsSmall MoleculesPeptidesPlasmid

Value Proposition Drug Delivery

Schematic of EXOPLEXs as a Drug Delivery Platform

This is our original idea when we first conceptualize EXOPLEXs2

EXOPLEXsAn Nanoparticle System

Enlarged ViewEXOPLEXsHigh drug loading capacity

Value Proposition Drug Delivery

As a Drug Delivery PlatformValue Proposition 1: High Drug Loading Capacity3

Targeted Drug Delivery

Tumour cellsTumour cells delivered with Anti-cancer Drug CargoIntravenous Injection

Tumour SiteDrug loaded EXOPLEXs

Targeting only Tumour Cells

Reduced Tumour size

Value Proposition Drug Delivery

As a Drug Delivery PlatformHow EXOPLEXs reach the target siteValue Proposition 2: Specific Targeting4

Therapeutics1Cosmeceutical / Dermatological Solutions2Transfection Kit for Difficult-to-transfect cell typesFor Research Use3What we thought5What we brainstorm during LLP

What we thought Market Segments

What we thought: Therapeutics as Original StrategyWhat we brainstormed at the start: Dermatological solutions and a transfection kit for research labs5

Research AssistantsPhDsPost DoctoralResearch FellowsPrinciple InvestigatorsCompaniesIndustry Liaison OfficeInterviews

MedicinePharmacyEngineeringChemistryCommercialInterviews (by field)6

Interviews Total 33

To find our customers, we went to interview various stakeholders6

Most Regulatory OversightLongest route to marketMost Requirement for dataLeast Regulatory OversightShortest route to marketLeast Requirement for data

Transfection Kit for Difficult-to-transfect cell typesFor Research Use

Cosmeceutical / Dermatological SolutionsTherapeutics123Continue in the backgroundNo interest

Beachhead Strategy

What we found Market Segments

We found that:Therapeutics should be still pursued as an over-arching strategy or goal2. No interest in EXOPLEXs as cosmetics3. Interest in EXOPLEXs as a transfection kit, especially for difficult-to transfect cell types

Why no. 3?Its the market segment that has the:Least regulatory oversightShortest route to marketLeast stringent demands for data7

Transfection Kit for Difficult-to-transfect cell typesFor Research Use

Cosmeceutical / Dermatological SolutionsTherapeutics123Continue in the backgroundNo interest

Beachhead Strategy

8Big PharmaTherapeutics CompanyCosmetic Companies

Laboratory End Users

Customers

From B2B to B2C

What we found Customers

Also the type of customers were very different for each market segmentOur original customers were the big pharma, but when we decided on pursuing the transfection kit strategy, our customers became the lab end users or those working in the research lab.8

EXOPLEXs

Genetic MaterialEnlarged ViewEXOPLEXsPlasmidsiRNAshRNA

Beachhead StrategyTransfecting Difficult-to-Transfect Cells

So what is this transfection kit for difficult-to-transfect cells all about?

Here, we describe how EXOPLEXs can be reformulated to transfer plasmids, or genetic material into cells.

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EXOPLEXs

Genetic MaterialEnlarged ViewEXOPLEXs

Beachhead StrategyTransfecting Difficult-to-Transfect Cells

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Single StepTransfection

Target cells(Eg. Fat cells, Blood Cells, Skin cells, Stem Cells)Cells expressing GFPEXOPLEXsWith plasmid

Transfecting Difficult-to-Transfect Cells

Beachhead StrategyTransfecting Difficult-to-Transfect Cells

* GFP: Green Fluorescent Protein

The Plasmid loaded EXOPLEXs enter the cells, instruct the cell machinery to produce more (or less) of a particular protein. In this case, we use Green Fluorescent Protein, hence the green fluorescence observed.11

12CAGR 7.1% from 2009 to 2016

What we found Market Sizing$183.6 MillionTransfection Reagents MarketTransfection Research Market

$30 Billion

Drug Delivery Market

$348 Million

1. Frost & Sullivan, 2010, Global Transfection Markets.2. Frost & Sullivan, 2016, Nanotechnology for Effective Drug Delivery

Is there a market for this?

We found that the overall market is growing at an increasing rate, a CAGR of 7%.

The Transfection Market (or target market) is $183 million, and the overall transfection research market is forecasted to be $348 million globally. In order to reach the huge Drug Delivery Market, we plan to start with our beachhead strategy, the transfection reagents market first.12

13Ease of UseEfficacy*size of circle: Cost EffectivenessEXOPLEXsLipofectamineEffectenePEIElectroporation

Combination of methods

Viral deliveryTransfection Research

What we found Competitive Landscape

Are we alone?Clearly no. But we do believe we have a competitive edge.

Here we constructed a 2x2 figure using transfection efficacy and ease of use as the main parameters. We found found our research that ease of use is one of the main reasons researchers look for. Our third parameter, depicted by the size of the circle, is cost-effectiveness.

This is where EXOPLEXs lie, the orange circle.13

14EXOPLEXs

Sales(Transfection Kit)Licensing

What we thoughtWhat we foundLicensing

EXOPLEXs

Lab End Users

Research Kit CompaniesResearch Kit Companies

What we found Business Model

Also, we learned that our business model may not necessarily be static.We started with the premise of doing licensing at first.

Then in the course of our interviews, we found the we can explore direct sales of the kit by ourselves.

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What we found Channels

Direct SalesDistributorshipsTimeCollaborationsEfficacy Data, PublicationsProduct SupportSmall scale manufactureBespoke servicesOEMSales force

So how do we plan to reach our customers?To target the innovators we plan to show them the efficacy data, and collaborate together to generate publications, as well as providing product support.Adding on would be the early adoptors, where we plan to sell in small scales directly to the labs as well as providing customized transfection services.To target the early majority and the rest, we hope to obtain a larger scale manufacturing, as well as a sales forces to push the product to the customers.15

Targeting & Minimal collateral damageImproved cell deliveryBiocompatibleAvert patent cliffUniversitiesPharma companiesTherapeutic companiesDrug Delivery companiesCosmetic companies

Production of:Efficacy dataSafety dataIntellectual propertyTrained laboratory usersRaw materialsEquipmentUniversitiesGovernment (Grants)In-house development of single chemotherapeuticIn-house development of EXOPLEXs as transfection agentIn-house development of EXOPLEXs as a cosmetic agentLicencing for other usesValue driven:R & D costsTrained personnel costsRaw materialsPatent developmentOutreach via publications, workshops and conferencesContinuous feedback with interested parties to fine tune EXOPLEXs platform to their use

Single sided niche market

B2B model

When we first started Business Model Canvas

This is our BMC when we first started.As you can see, we tried to target almost everyone except the lab end users. So this is a case showing what we have in mind may not always be true in the real world.16

Key PartnersKey ActivitiesValue PropositionKey ResourcesCustomer RelationshipsChannelsCustomer SegmentsCost StructureRevenue Streams

PublicationsEfficacy dataComparison data

Transfecting Difficult-to-Transfect cellsImproved user experienceIncreased research efficiency

Co-development/ collaboration with innovators & early adoptersDirectAcademic and research labs focusing tissue labsPrimary cell linesSkin, fat, neurons, stem cells

Intellectual PropertyTrained lab personnelKeep costs down, asset light by staying affiliated to NUSCurrently exploring other revenue streams, ie direct sales and co-development with other companies.

After 10 weeks Business Model Canvas

And after 10 weeks, this is our BMC.Our Value Proposition is to transfect better difficult to transfect cells, as well as providing a better user experience.We plan to do it directly to the customers, and this is our lab end users. These include those in tissue and cell labs, such as the Pis, post docs, Phds.We Plan to keep our costs low and stay assets light, by staying affiliated to NUS.We also intend to explore our revenue streams beside direct sales such as co-development with other companies, as well as keeping the option of licensing should such opportunities arises. Therefore it is extremely important that we keep our customer relationships with the lab users open and on-going, through active collaborations and co-development.

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18Transfecting difficult to transfect CellsDrug DeliveryClinical ApplicationsGene TherapyPeptide-based DeliverySmall Molecule DeliveryBeachhead StrategyRevenue Generation Direct salesLicensingDistributorshipsLong Term StrategyRevenue Generation Licensing Co-development2016: $183.6 Million12020: $30 Billion1. Frost & Sullivan, 2010, Global Transfection Markets.2. Frost & Sullivan, 2016, Nanotechnology for Effective Drug Delivery

Time (Years)Difficulty

Roadmap for the Future

This is our roadmap for the future.Our beachhead strategy is coming up with a kit for the difficult-to-transfect cells market.Long term, we plan to enter the drug delivery market, where there are several applications possible. Why drug delivery? Because while our beach head strategy has a market size of $183 million, the drug delivery market is estimated to be a huge $30 billion.18

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What we have nowWhat we will do nextProvisional PatentEfficacy dataMore R & D for MVPPublicationsFind potential Collaborators (Labs)Applications for Grants (POC)

Next Steps

What's next?Currently, we have a provisional patent and some preliminary data.

What we will do next is More R & D for a MVP to beta test on our next door labPublish publish publishFind potential collaboratorsApply for more grants19

20Wei Jiang GOH

Principle Investigator

Department of PharmacyEntrepreneurial LeadPhD-MBA StudentDepartment of PharmacyTeam MemberPhD StudentDepartment of PharmacyA/P Giorgia PASTORINShui ZOU

The TeamTeam Mentor

Dr Ju Wei Wong

This is our team20

21Thank You

EXOPLEXs

Thank you21

EXOPLEXsStrategyTransfection KitDifficult-to-transfect cellsDrug delivery platformSmall moleculesPeptidesGenetic materialSmall Scale ManufacturingLicensingLicensingCo-DevelopmentDirect SalesSales & DistributorshipResearch Lab End UsersResearch Reagent CompaniesGenerics CompaniesDelivery to difficult to reach cellsNeurodegenerative diseasesDiabetesPartnership with emerging biotech companiesPotential NDAsSearch for off-patent drug for averting patent cliffPharma CompaniesBiotech CompaniesStrategyRevenue ModelCustomer*Beachhead strategy2020: $30 Billion2016: $183.6 Million*Frost & Sullivan

Supplementary slide22