CNS-related diseases (in general)

Bioneer has over 100 publications in CNS research, including collaborations with Lundbeck and Johnson & Johnson.

We offer a broad portfolio of CRISPR gene-edited iPSC cell lines that can be used to study CNS diseases beyond Parkinson’s and Alzheimer’s disease.

We work closely with you to design experiments, select the right cell lines, and adapt our models to your disease or mutation of interest, making sure the solutions fit your project and timelines.

These models can be applied to a range of CNS-related diseases, including neurodegenerative conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS).

The lines are based on the same robust platform used for our Parkinson’s Disease and Alzheimer’s disease models, with differentiation and mutations tailored to the disease of interest.

other CNS-related diseases

Many of our iPSC lines have been thoroughly characterized and can be adapted to support your specific drug discovery questions. This makes them suitable for investigating genetic disease mechanisms, cellular function, and drug effects.

Our progranulin heterozygous and homozygous knock-out cell lines can, for example, be used to model frontotemporal dementia and ALS, with functional studies in iPSC-derived neurons, astrocytes, and microglia. Some of the read-outs could inform you about lysosomal function and progranulin release related to your drug candidates.

CNS-related diseases in general

Key benefits of our CNS disease models and assays

  • Human iPSC-based, disease-relevant neuronal and glial systems
  • Flexible model design adapted to disease biology and genetic context
  • Functional and translatable readouts across CNS indications adapted to your drug discovery needs

Applications

  • Drug discovery and compound profiling
  • Target validation and mechanistic studies
  • Preclinical support using human-relevant in vitro data

Relevant iPSC cell lines

Listed below are examples of CRISPR gene-edited iPSC cell lines that can be used to study genetic effects linked to different CNS-related diseases.

Background cell line Gene  modification/insertion Disease relevance or biology
BiONI010-C SNCA A53T dox inducible PD
BiONI010-C/UKBi011-A/BIONi037-A APOE2/2 AD
BiONI010-C/UKBi011-A/BIONi037-A APOE3/3 AD
BiONI010-C/UKBi011-A/BIONi037-A APOE4/4 AD
BIONi010-C with APOE 2/2, 3/3 and 4/4 APOE Christchurch AD
BIONi010-C-54/UKBi011-A/BIONi037-A-4 APP swedish AD
BIONi010-C-54/UKBi011-A/BIONi037-A-4 APP London + Swedish AD
BIONi010-C-54/UKBi011-A/BIONi037-A-4 APP London AD
BIONi010-C APP KO AD
BIONi010-C ATXN1 KO Spinocerebellar ataxia type 1 (SCA1)
SA001 ATXN2 KO Spinocerebellar ataxia type 1 (SCA2)
BIOONi010-C PSEN1 – E280A AD
BIONi010-C GRN – KO FTD/ALS
BIONi010-C GRN – R493X FTD/ALS
BIONi010-C SARM1 – KO Pain
BIONi010-C 2N4R Tau AD
BIONi010-C 2N4R Tau – P301L AD
BIONi037-A-8 KCNT1 G288S PD
BIONi010-C/ BIONi037-A LRRK2 – G2019S PD
BIONi037-A TMEM117 M393T AD
BIONi037-A TMEM117 Q65P AD
BIONi010-C TREM2 – D87N AD
BIONi010-C TREM2 R47H AD
BIONi010-C TREM2 R62H AD
BIONi010-C TREM2 T66M AD
BIONi010-C TREM2 KO (BIONi010-C-17) AD
BIONi010-C CD33 – KO AD
BIONi010-C CD33-Exon2 deletion AD
BIONi010-C SGSH KO Sanfilippo syndrome A
SIGI001-A NLRP3 KO Autoinflammatory disorders
SIGI001-A MAPT KO AD
BIONi010-C-13 UBE2M KO Inflammation
BIONi010-C-13 UBE2F KO Inflammation

 Read more about our BIONi10-C cell lines here.

Our CNS leadership

Dept. Head of Cellular Engineering and Disease Models

Bjørn Holst,

Dept. Head of Cellular Engineering and Disease Models

Kenneth Thirstrup, CNS assay development

Kenneth Thirstrup,

R&D Manager, CNS Assays

Explore related CNS disease models:

Related CNS biology:

  • Neuroinflammatory diseases/ Neuroinflammation

Other CNS capability:

  • Pain-related studies

Talk with our team

We’re happy to discuss how our iPSC lines and neurodegenerative disease models can support your specific research and drug discovery needs.

Business Development Manager, CNS

For further information please contact  Business Development Manager Jacob Mathias Bech on jmb@bioneer.dk or +4523202576

Business development manager, CNS

For further information please contact  Business Development Manager Jacob Mathias Bech on jmb@bioneer.dk or +4523202576