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.

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.

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

Bjørn Holst,
Dept. Head of Cellular Engineering and Disease Models

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.

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

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

