Name: Advancing global health: a journey of breakthroughs and collaboration
Uploaded: 2023-08-04T05:31:31.968Z
Duration: 558.09
Description: In this interview, Dr Marthe D’Ombrain, Head of Global Research External Innovation, discusses CSL groundbreaking work in biotherapeutics. Learn how they are improving treatment for hemophilia B patients with innovative therapies, including gene therapy.

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The third pillar that we

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believe is really required

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to achieve the translation

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of outcomes is skills.

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We need to ensure that

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we are upskilling.

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Hello, and thank you

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very much for having me

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here for this interview.

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My name is Marthe

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D’Ombrain, and I head

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up CSLs Global Research

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External Innovation team.

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And what my team essentially

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does is look across the

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globe for novel early

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stage opportunities that

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we think could be the next

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generation of therapies

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for patients living

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with unmet medical need.

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We are really happy to share

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some of the advances we are

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making in Biotherapeutics.

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So let me use hemophilia

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and what we're doing

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for hemophilia B

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patients, as an example.

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So, hemophilia B is a clotting

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disorder, where patients have,

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a, a defective, factor nine

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gene, and an inability to clot

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blood, appropriately after

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injury or surgery, or it can

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also just happen spontaneously

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and so it can really, cause

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a terrible quality of life

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for patients living with

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hemophilia B and, and clotting

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disorders in general and CSL

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has been making therapies,

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plasma derived therapies for

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hemophilia B patients for

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decades, where we, purify the

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hemophilia B protein, factor

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nine protein from plasma, and

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convert that into a medicine

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so that patients can

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replace the fact that it's

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missing and have their blood

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clotting appropriately.

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Now, one of the things

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that really holds back

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therapy for patients with

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hemophilia B is that plasma

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derived therapies needs to

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be given really frequently.

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So two or three times

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a week through an

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intravenous infusion.

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And so, as you can imagine,

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that's very challenging

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and it's very disruptive

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to quality of life.

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So a few years ago we

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generated a new product,

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which is a recombinant

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version of factor nine

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and it's long acting, it's

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fused to albumin and that

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has enabled hemophilia B

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patients to now have therapy

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every two weeks and in some

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cases every three weeks.

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So really, improving the

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quality of care, without

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compromising the efficacy.

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But we didn't stop there.

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We're all about innovation at

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CSL and we are very motivated

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by patients and, and improving

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the lives of patients.

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And so recently we

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partnered with uniQure to

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bring the very first gene

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therapy for he hemophilia

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B patients two patients

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and we are very proud to

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say that that has now been

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approved, in the US and in

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Europe and our first patient

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has been dosed, with this

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therapy in the us so it

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provides hemophilia patients

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with now another option,

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a potentially curative

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option, where, no additional

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prophylaxis is, is required.

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I think one of the themes

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that really emerged

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from the pandemic and

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our experience in the

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pandemic was collaboration.

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Collaboration is one of

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CSLs core values, and it

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really underscored the

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pillar of our response.

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To the pandemic.

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I think everyone in Australia

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will, will remember and

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know that we pivoted our

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manufacturing really quickly

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to be able to make and

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deliver 50 million doses

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of Covid vaccine to the

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Australian public and our

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neighboring countries.

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And that was a, a really

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incredible effort, by

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our people at C S L.

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It wasn't just a matter

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of pushing a button and

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being able to pivot our

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manufacturing processes.

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There was a lot of change

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and adjustment that had to

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be done, but we were able to

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do that in a nimble and agile

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way and, respond to the needs

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of the Australian people.

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And in terms of future

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preparedness, we are really

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proud to be building a

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new $800 million, vaccine,

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innovative vaccine, and

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anti-venom facility.

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So again, really shoring

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up Australia's ability

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to be able to make,

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vaccines both seasonal.

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Seasonal and pandemic vaccines

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on shore outside of vaccines.

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We also, collaborated with a

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number of other pharmaceutical

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companies to try and

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develop a covid hyperimmune,

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during the pandemic, and

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we also partnered with the

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University of Queensland.

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To, try and develop, an

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Australian grown vaccine.

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And, and you may recall

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that that didn't work, but

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I think it really shows

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CSLs commitment to be agile,

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to be nimble, to respond,

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appropriately and support

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the Australian public.

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You know, if you're

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not trying to digitize.

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All the processes you

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can today, then you're

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really not innovating.

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And so this is something that

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we are focused on in many

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areas across our business and

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our business units, in terms

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of bypro, and manufacturing.

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So, recently,  there was

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an announcement of a new

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AICARC research hub for

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digital Bioprocessing.

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And so we have partnered

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with the University of

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Melbourne, and a number of

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other companies, around this.

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This hub for,

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bioprocessing research.

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And what this hub, aims to

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achieve is to really look

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for, ways that bioprocessing

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and manufacturing can be

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improved, particularly

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through digitization,

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developing new processes,

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but also predictive models.

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That will enable us to then

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implement those outcomes

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from that research hub

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into our own processes to

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help with automation and

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digitization, and ultimately

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streamline our processes.

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So that's just one example

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of how we are doing that.

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Certainly at C S L, we

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really believe, again, in

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collaboration, it has to

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be a collaborative effort

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that really drives the

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translation of discoveries

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through to commercialization

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and access for patients.

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So in Australia, you know,

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we, we need to ensure that

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we have funding available

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to advance, not only for

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discovery research, but also

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for translational research.

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And I think we've

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made some really good

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headway in that area.

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In recent times, there's

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been a number of new funds,

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launched and available now

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for translation and, and we

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need to also make sure we're

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protecting investment in

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discovery research as well.

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Infrastructure is

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another key piece.

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We need to ensure that we

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are continuing to invest

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in advanced manufacturing

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and onshore infrastructure

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and capabilities to

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support the development of

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therapeutics and vaccines.

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And I, and I guess

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I've touched on some of

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those ways that C S L

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is continuing to invest.

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We've invested

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over $2 billion.

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In infrastructure in Australia

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alone over the past couple

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of years, I talked about

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the $800 million innovative

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vaccine and anti-venom

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facility in Tamarine.

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We also invested $600

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million in an expansion of

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our plasma fractionation

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facility in Broad Meadows,

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and we've just opened our

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new state-of-the-art, global

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headquarters on Elizabeth

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Street North, in Parkville.

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So in addition to

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infrastructure and

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capabilities, the third

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pillar that we believe

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is really required to

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achieve, the translation

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of outcomes is skills.

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We need to ensure that we

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are upskilling, our, the

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people in Australia who can

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not only make discoveries,

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I think we've got fantastic

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discovery scientists, but

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the people who can run the

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companies once we, you know,

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translate those discoveries

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and spin them out into a

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biotech company, for example.

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So we need those.

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SW C-suite professionals,

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who can do that.

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And again, CSL is really

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trying to get behind a

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number of initiatives, to

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ensure that we are developing

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the future, in Australia.

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And so we've just, announced

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a partnership with the

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University of Melbourne

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and the Walton Eer Hall

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Institute, to establish

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an incubator within CSLs

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Global Head headquarters.

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This is called, Juma

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Bio Incubator, with

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investment also from

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Breakthrough Victoria.

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And it's being operated

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by Karta innovation.

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So again, fantastic

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collaborative effort that

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really aims to provide an

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ecosystem and a community

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that enables, scientists

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and founders who are

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wanting to translate and

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commercialize discoveries.

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A place to come where they

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can learn and mingle with C

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cell scientists, people who do

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deliver solutions to patients

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day in, day out and cross

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pollinate and share ideas.

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We also, invest heavily

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in training and education.

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So for example,  we believe

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strongly in industry exchange

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and we've in my team as

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well, but also across CSL,

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we've had a number of,

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scientists and business

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development professionals.

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Come into, our organization

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and, be seconded there so

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that they can then take those

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industry learnings back out

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into the sector and one of

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the fantastic programs that

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has supported that is the

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Ready Fellowship program

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from,  MTP Connect and MRFF.

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We also support, you know,

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earlier programs such as

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the undergraduate Research

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Opportunities program,

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and we also provide,

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fellowships such as the

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CSL Centenary fellowships.

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So I think, there are

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many ways that we can all

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work together to achieve

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this, but if I had to

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sum it up in one word, it

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would be collaboration.

Expert Insight

In this interview, Dr Marthe D’Ombrain, Head of Global Research External Innovation, discusses CSL groundbreaking work in biotherapeutics. 

Learn how they are improving treatment for hemophilia B patients with innovative therapies, including gene therapy. 