Research Valorization Business Development

Collaboration and business

The Business Development team helps our researchers in collaborations with external parties, protecting and exploitation of inventions, negotiating deals and setting up companies.

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Collaboration and business

Besides stimulating and negotiating research collaborations, we support the creation of spin-off companies with technologies from Radboudumc by evaluating business plans, protecting IP rights, negotiating license agreements, searching for initial funding and initiating contact with candidate CEOs and other experts.

We recently strengthened our collaboration with Janssen Pharmaceutica NV, Janssen-Cilag B.V. and Janssen Vaccines & Prevention B.V  to bring high quality innovative academic research faster to the patient and we concluded a partnership with Viiv Healthcare for identification of novel targets in HIV.

Through its subsidiary UMC St. Radboud Holding, Radboudumc takes an interest in these spin-off companies and keeps track of their development. The Holding has 19 spin-off companies in which 153 FTE are employed, and in which to date EUR 44M has been invested.

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Secretariat Business Development

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Licensing opportunities


Antisense oligonucleotides for the treatment of inherited deafness DFNA9

We developed AONs that may be used in therapy for inherited deafness.

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Antisense oligonucleotides for the treatment of inherited deafness DFNA9

DFNA9 is a relatively common form of adult-onset, autosomal dominantly inherited, progressive hearing loss. DFNA9 is caused by mutations in the COCH gene, and leads to complete deafness by the age of 50-70. DFNA9 patients furthermore suffer from severe balance loss, hampering their daily movements. Dominant COCH mutations result in the formation of cytotoxic protein aggregates, consisting of both mutant and normal COCH proteins, that interfere with hearing and balance function.

Currently, no effective treatment is available to prevent or delay the loss of hearing and balance problems in DFNA9 patients. Antisense oligonucleotides (AONs) are small synthetic single-stranded DNA/RNA molecules that can be used target pre-mRNA for degradation. In a dominantly inherited disorder such as DFNA9, they can be used to specifically degrade mutant COCH transcripts and/or block the translation of mutant COCH protein thus preventing the formation of cytotoxic protein aggregates.

To develop AONs with high specificity for the mutant allele, we first used long-read haplotype sequencing to identify all mutant-allele specific variants that can serve as therapeutic targets for the majority of patients carrying the c.151C>T(p.P51S) and c.263G>A (p.G88E) founder alleles.

We obtained proof-of-concept that both a disease-causing variant, and mutant allele-specific variants, can be used as potent discriminating variants to specifically decrease mutant COCH transcript levels, without interfering with wildtype transcript levels. An example of this is provided in figure 1.

Applications

  • Ophthalmology
  • Stargardt disease
  • Antisense oligonucleotide therapy

Development status: Proof of concept

  • We obtained proof-of-concept in cells expressing mutant and wildtype COCH alleles, and are working on the preclinical lead optimization in preparation of a clinical use.
  • Additional mutant allele-specific variants on the c.151C>T(p.P51S) and c.263G>A (p.G88E) founder alleles will be analyzed for their eligibility as targets for AON-based therapy.

Opportunity

  • We offer a licensing opportunity of the patent covering the described invention.
  • We are interested in collaboration and/or co-development of the invention.

Contact

For more information contact us through businessdevelopment@radboudumc.nl.


Expandable foetal plug A solution for the Achilles heel of foetal endoscopic surgery

We developed an advanced collagen plug (medical device) that closes the membranes at the endoscopic entry point in fetoscopy.

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Expandable foetal plug A solution for the Achilles heel of foetal endoscopic surgery

Minimal invasive foetal surgery using endoscopy is a promising medical technology to treat children before birth, e.g. children with rare diseases like twin to twin transfusion syndrome. During endoscopy, the maternal abdominal wall, uterus, and membranes surrounding the foetus are punctured. This introduces a risk for leakage of amniotic fluid and a risk of up to 40 of membrane rupture. Ruptures induced by foetal endoscopic surgery is a strong trigger for premature birth, infection and maternal sepsis and is considered the “Achilles heel” of foetal endoscopic surgery. Until now, there is no satisfying solution for this problem.

We developed an advanced collagen plug (medical device) that closes the membranes at the endoscopic entry point in fetoscopy, thereby preventing amniotic fluid leakage and associated severe complications The plug is designed to expand upon placement, seal the membranes, and allow regeneration of amniotic/chorionic tissue, hence preventing rupture. The plug is crimped before application allowing the use of minimally invasive surgical instruments and deploys when applied into the defect. This invention describes the first plug with shape memory (introduced by the creation of local hydrophobic patches) which gives the product its unique features and added value.

Applications

  • Neonatology
  • Foetal surgery
  • Premature birth
  • Pregnancy

Development status

  • Functional design is ready and tested in several animal models with great results (TRL 3 ­> experimental proof of concept)
  • Patent pending (priority date December 31st 2020)
  • Funding has been granted to further develop and clinically validate the expandable collagen plug

Opportunity

  • We offer a licensing opportunity for the patent covering the described invention
  • We are interested in collaboration and/or co-development

For more information contact us through businessdevelopment@radboudumc.nl.


Immuno­combination therapy for cancer

Novel small molecule that modulates the immunesystem to fight cancer.

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Immuno­combination therapy for cancer

Tumor cells express high levels of sialic acid sugars on their surface. Sialic acids suppress the immune system in the tumor microenvironment. Blocking tumor sialic acid expression slows tumor growth/metastasis and boosts immune therapy efficacy. We developed a potent (~1µMEC50) small molecule inhibitor of the endogenous sialic acid biosynthesis. After passive transport over the cell membrane the compound is metabolized into the active inhibitor. This compound remains in the target cell and cannot enter other cells after cell death, thereby limiting its effects to the target cell alone.

Applications

  • Therapeutics for treating and preventing cancer. - Activator of immune cells that are used for cell-therapeutic applications.

Development status

Patent application has been filed. In vivo efficacy in slowing tumor growth and metastasis was demonstrated with a lesser potent, inhibitor. The current lead has an improved potency based on the C-5 substituents.

Opportunity

  • Out-licensing
  • Development collaboration

For more information contact us through businessdevelopment@radboudumc.nl.


Intranasal vaccine against Streptococcus pneumoniae

This vaccine offers a broad protection against Streptococcus pneumoniae infections.

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Intranasal vaccine against Streptococcus pneumoniae

Discovery of novel protein antigens using whole genome based antigen discovery tools (Tn-Seq and Genomic Array Footprinting). Antigen fragments are delivered using outer membrane vesicles (OMVs), which induce strong immune responses and protection against carriage.Administration is intranasal and strong protection is achieved in a mouse mode

Applications

  • Broad protection against Streptococcus pneumoniae infections in children and elderly
  • Reduction of carriage > reduction of transmission
  • Affordable needle free and easy to apply: intranasal delivery

Development status

Patent filed and 2 patents pending. We have proof of concept in mice and are working on lead optimization in pre-clinical studies and in parallel work on the scale up of manufacturing under GMP. In collaboration with experts we are currently testing spray devices for delivery of the OMV containing pneumococcal antigens. The clinical phase I study is in preparation.

Opportunity

  • Out-licensing
  • Development collaboration from clinical phase IIa

For more information contact us through businessdevelopment@radboudumc.nl.


Genetically matched immortalized muscle cell cultures of FSHD patients

This cell line can be used in tissue culture studies into the causes of DUX4 expression and is well suited for drug screening in FSHD.

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Genetically matched immortalized muscle cell cultures of FSHD patients

Field of Use

Drug screening

Key benefits for end user

  • Well characterized immortalized muscle cell cultures representing key pathophysiological features of facioscapulohumeral dystrophy
  • Derived from mosaic FSHD1 patients: genetically matched clonal cell lines with or without FSHD1 mutation

Background

  • Facioscapulohumeral dystrophy (FSHD) is one of the most common muscular dystrophies world-wide (estimated incidence 1:8,500 – 1:15,000);
  • FSHD is characterized by progressive and irreversible loss of facial and upper extremity muscles;
  • No therapy is available for FSHD;
  • FSHD is caused by inappropriate expression of the transcription factor DUX4 in skeletal muscle;
  • In >90% of patients inappropriate DUX4 expression is the consequence of the contraction of the D4Z4 macrosatellite repeat on chromosome 4 to a size of 1-10 units (FSHD1);
  • Half of de novo D4Z4 contractions occur after fertilization resulting in somatic mosaicism: muscles are composed of a mix of genetically affected and unaffected myonuclei.

Invention

Genetically matched affected and unaffected FSHD muscle cell lines

Taking advantage of the high frequency of somatic mosaicism in de novo FSHD1, we created pairs of genetically matched immortalized muscle cell lines prior and after D4Z4 contraction from independent mosaic FSHD1 muscle biopsies.

  • Immortalization of primary FSHD1 muscle cell cultures by stable expression of hTERT and Cdk4;
  • Clonal expansion and characterization of genetically affected and unaffected immortalized FSHD1 muscle cells.

Development status

  • Fundamental research: Validated for fundamental tissue culture studies into the causes and consequences of DUX4 expression.
  • Applied research: Validated for drug screening studies into compounds that reduce or prohibit DUX4 expression, or its molecular and cellular consequences.

Applications

Screening

  • Due to their immortalization and extensive genetic and molecular characterization the genetically matched pairs of FSHD muscle cell cultures are well suited for drug screens:
  • These cell lines have demonstrated use in small molecule drug screens for FSHD (ref 2);
  • These cell lines have demonstrated use in antisense oligonucleotide therapy screens for FSHD (ref 3);
  • These cell lines have demonstrated use in epigenetic modifier screens for FSHD (ref 4).

Opportunity

Outlicensing, collaboration.

Patents and key publications

  1. Krom YD et al., Generation of isogenic D4Z4 contracted and noncontracted immortal muscle cell clones from a mosaic patient: a cellular model for FSHD. Am J Pathol. 2012 Oct;181(4):1387-401. doi: 10.1016/j.ajpath.2012.07.007.
  2. Rojas LA et al., p38α Regulates Expression of DUX4 in a Model of Facioscapulohumeral Muscular Dystrophy. J Pharmacol Exp Ther. 2020 Sep;374(3):489-498. doi: 10.1124/jpet.119.264689.
  3. Lu-Nguyen N et al., Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model. Hum Mol Genet. 2021 May 13:ddab136. doi: 10.1093/hmg/ddab136.
  4. Campbell AE et al., BET bromodomain inhibitors and agonists of the beta-2 adrenergic receptor identified in screens for compounds that inhibit DUX4 expression in FSHD muscle cells. Skelet Muscle. 2017 Sep 4;7(1):16. doi: 10.1186/s13395-017-0134-x

Contact

For more information contact us through businessdevelopment@radboudumc.nl.

Business development experts