Mosaic Partner Survey

Executive Summary

Mosaic is developing the AI-Driven Multiplexed Personalized Assay (AMPA) Platform, an open-innovation system designed to help researchers and drug developers model patient heterogeneity in sporadic neurodegenerative diseases such as ALS.

The goal of this survey is to better understand how potential partners and collaborators currently approach preclinical discovery and development, what gaps and unmet needs exist, and how Mosaic can best design tools, assays, and analytics that accelerate your programs' success.

Your feedback will directly shape the AMPA platform’s features, services, and collaboration model. Responses will remain confidential and will be summarized in aggregate to guide the design of shared research and pilot programs.

Estimated time to complete: 5 minutes

Section 1: Background

Question Title

* 1. Which best describes the organization you work for?

Nonprofit / Gov't Research Sponsor

Venture Capital Firm / Accelerator

CRO / CDMO

Academic Research Institution

Biotech / Pharmaceutical Company

Other (please specify)

Question Title

* 2. Which therapeutic areas or disease models are you most actively involved in?

Parkinson’s Disease

FTD

ALS

Alzheimer’s Disease

Other (please specify)

Question Title

* 3. How would you describe your organization’s current focus within the preclinical pipeline? (select all that apply)

Target identification and validation

Assay development

High-throughput screening

Mechanistic studies / biomarker discovery

IND-enabling studies

Other (please specify)

Question Title

* 4. Which of the following best describes your role? (select all that apply)

Discovery / Preclinical Scientific Lead

Translational or Clinical Development Lead

Program, Portfolio or Partnerships Manager

Executive Leadership (CSO, VP, etc.)

Other (please specify)

Section 2: Current State and Needs

Question Title

* 5. Which types of models, tools, or platforms do you currently use for preclinical or translational research? (select all that apply)

Animal models

iPSC-derived neuron or glial models

CRISPR-engineered lines

AI / in silico modeling

Organoids

Other (please specify)

Question Title

* 6. How do you currently address patient heterogeneity (e.g., sporadic vs. familial, molecular subtypes) in your preclinical work?

We rely primarily on different monogenic models

We use multiple models representing diverse subtypes, including sporadic cases

We have limited ability to model patient diversity currently

We stratify samples based on molecular biomarkers

Question Title

* 7. What are your biggest challenges in developing or testing assets for sporadic neurodegenerative diseases? (select up to 3)

Lack of reproducibility across labs

Inability to model patient heterogeneity

Limited access to patient-derived materials and models

Poor translation from preclinical to clinical outcomes

Difficulty in validating mechanisms of action

Limited availability of relevant disease models

Limited availability of appropriate Biomarkers

None of the above - I'm satisfied with the state of the field

Other (please specify)

Question Title

* 8. What barriers prevent you from testing or validating compounds across diverse patient subgroups? (select all that apply)

Lack of scientific consensus on subgroup classification

IP or collaboration constraints

Cost and complexity

Data integration / analysis limitations

Access to models

Lack of standardized tools

Other (please specify)

Question Title

* 9. How do you currently evaluate mechanism of action or biomarker associations before entering clinical studies? (select all that apply)

Phenotypic screens

Target-based assays

Collaborations with CROs or academic labs

Omics profiling (RNA-seq, proteomics, etc.)

Bioinformatics / AI analysis

Other (please specify)

Question Title

* 10. In your experience, where do current tools, platforms, or service providers fall short of meeting your preclinical research needs?

Section 3: Drug Development Strategies
Please indicate how strongly you agree or disagree with the following statements.

Question Title

* 11. Although neurodegenerative diseases are highly heterogeneous, drug development focused on disease pathways identified with monogenic models is likely to remain a more promising path to trial success than targeting subtypes.

Strongly disagree

Disagree

Neutral

Agree

Strongly agree

Strongly disagree

Disagree

Neutral

Agree

Strongly agree

Question Title

* 12. Like cancer, neurodegenerative diseases require subtype-specific therapies. Precision drug development offers a more promising path to trial success – but the field needs better preclinical tools to make this possible.

Strongly disagree

Disagree

Neutral

Agree

Strongly agree

Strongly disagree

Disagree

Neutral

Agree

Strongly agree

Question Title

* 13. Differences in patients’ genetics contribute to clinical heterogeneity and variation in drug response, yet most available disease models do not capture this diversity.

Strongly disagree

Disagree

Neutral

Agree

Strongly agree

Strongly disagree

Disagree

Neutral

Agree

Strongly agree

Section 4: Feedback on the Mosaic AMPA Platform

In this section, we’d like to ask about the platform we’re developing. Please read this brief description and answer the questions below.

About the Mosaic AI-Driven Multiplexed Precision Assay Array (AMPA) Platform

Mosaic Neuroscience’s AMPA™ platform enables “Phase 0 clinical trials in vitro” – allowing biopharma partners to identify responsive patient subgroups before costly human trials. AMPA integrates patient-derived neuron models, high-throughput drug screening and AI-driven analytics to determine drug-response patterns across diverse forms of sporadic neurodegenerative disorders. For R&D teams, the platform delivers deeper insights on mechanisms and biomarkers, enabling earlier de-risking, targeted trials, and faster approvals of precision therapies for the 90% of patients with sporadic disease.

Question Title

* 14. Which components of the Mosaic AMPA Platform are most relevant or appealing for your work? (select all that apply)

Preclinical in vitro trials integrating molecular and phenotypic data

Large language model integration for literature-augmented hypothesis generation

Standardized iPSC panels validated across labs

High-throughput compound testing across heterogeneous patient lines

Other (please specify)

Question Title

* 15. Which potential features would create the most value for your research or development efforts? (select up to 3)

AI-driven patient stratification and biomarker discovery

Purchase pre-plated sporadic iMNs to run in-house

Integration of multi-omics data for mechanism analysis

AI-powered data interpretation and hypothesis generation

Networked collaboration and data sharing among labs

Access to standardized sporadic ALS patient-derived cells

Multiplexed drug testing across multiple ALS subtypes

Other (please specify)

Question Title

* 16. How important would standardized patient-derived models of diverse forms of sporadic ALS, verified across multiple labs/CROs, be for your organization’s reproducibility and collaboration goals?

Extremely important

Very important

Moderately important

Slightly important

Not important

Extremely important

Very important

Moderately important

Slightly important

Not important

Section 5: Desired Platform Capabilities

Question Title

* 17. Which disease phenotypes would be most valuable to model or measure? (select all that apply)

Axonal function / transport

TDP-43 loss of function

Autophagy/ Mitophagy

Extracellular vesicles

Nuclear pore function

TDP-43 localization

Neurite growth

Neuromuscular junction function

Sensitivity to cellular stresses

Expression of cryptic exons

NfL secretion

Electrophysiological properties

Oxidative Stress

Glial pathology

Other (please specify)

Question Title

* 18. Given the ability to test assets in models reflecting diverse patients, in what ways would you want to use the platform? (select all that apply)

Screening across multiple subtypes (“mosaic”) to assess differential response

Identification of novel mechanisms of action

Compound library screening across single ALS subtypes

Discovery of new disease pathways or targets

Toxicity prediction studies

Lead optimization studies

Other (please specify)

None of the above

Question Title

* 19. How would you prefer to engage with the AMPA platform?

As a service performed by Mosaic

By purchasing the AMPA system to run in-house

Both options would be of interest

Not sure yet

Section 6: Appetite to Partner or Support

Question Title

* 20. Please provide the name of your organization.

Question Title

* 21. As we validate and test the platform, would you be interested in participating as a beta tester?

Yes, very interested

Possibly, depending on timing and scope

Not at this time

Question Title

* 22. Would you be willing to sign a non-binding Letter of Support (LOS) expressing interest in collaborating on developing preclinical models of sporadic ALS (such as AMPA)?

Yes

Possibly – I’d like to learn more first

Not at this time

Question Title

* 23. In 2026, the Act for ALS will be up for renewal. We are considering advocating for the new legislation to include an SBIR/STTR program to provide competitive, non-dilutive grants ($350k-2M) to ALS biotechs with promising therapeutic ideas. Would such a program help your organization develop drugs faster?

Yes — Access to early-stage capital is one of our biggest barriers, and this program would directly accelerate our ALS therapeutic development.

Yes — It would significantly reduce financial risk and shorten timelines for one or more ALS programs we otherwise cannot fund.

Yes — It would allow us to pursue promising preclinical ideas currently on hold due to limited funding for ALS discovery work.

Potentially — Impact would depend on eligibility, timing, and whether funds can support high-risk preclinical discovery and model development.

Not directly — but it would strengthen our ecosystem by funding partners whose innovation we rely on (CROs, academic spin-outs, tool developers).

No — We do not conduct preclinical ALS therapeutic development.

Other (please specify)

Section 7: Collaboration and Next Steps

Question Title

* 24. Would you be interested in participating in a virtual industry workshop to discuss shared challenges and opportunities in preclinical modeling for sporadic neurodegenerative diseases?

Yes – I’d like to participate

Possibly – please keep me informed

No, not at this time

Question Title

* 25. If yes, please provide the contact details for participating in the virtual industry workshop (optional):

Name:

Email:

Thank You!

Your feedback will directly inform Mosaic’s AMPA platform development roadmap and our collaboration model. We appreciate your time and partnership in shaping the future of preclinical neuroscience research.

Mosaic Partner Survey

Executive Summary

Question Title

* 1. Which best describes the organization you work for?

Question Title

* 2. Which therapeutic areas or disease models are you most actively involved in?

Question Title

* 3. How would you describe your organization’s current focus within the preclinical pipeline? (select all that apply)

Question Title

* 4. Which of the following best describes your role? (select all that apply)

Question Title

* 5. Which types of models, tools, or platforms do you currently use for preclinical or translational research? (select all that apply)

Question Title

* 6. How do you currently address patient heterogeneity (e.g., sporadic vs. familial, molecular subtypes) in your preclinical work?

Question Title

* 7. What are your biggest challenges in developing or testing assets for sporadic neurodegenerative diseases? (select up to 3)

Question Title

* 8. What barriers prevent you from testing or validating compounds across diverse patient subgroups? (select all that apply)

Question Title

* 9. How do you currently evaluate mechanism of action or biomarker associations before entering clinical studies? (select all that apply)

Question Title

* 10. In your experience, where do current tools, platforms, or service providers fall short of meeting your preclinical research needs?

Question Title

* 11. Although neurodegenerative diseases are highly heterogeneous, drug development focused on disease pathways identified with monogenic models is likely to remain a more promising path to trial success than targeting subtypes.

Question Title

* 12. Like cancer, neurodegenerative diseases require subtype-specific therapies. Precision drug development offers a more promising path to trial success – but the field needs better preclinical tools to make this possible.

Question Title

* 13. Differences in patients’ genetics contribute to clinical heterogeneity and variation in drug response, yet most available disease models do not capture this diversity.

Question Title

* 14. Which components of the Mosaic AMPA Platform are most relevant or appealing for your work? (select all that apply)

Question Title

* 15. Which potential features would create the most value for your research or development efforts? (select up to 3)

Question Title

* 16. How important would standardized patient-derived models of diverse forms of sporadic ALS, verified across multiple labs/CROs, be for your organization’s reproducibility and collaboration goals?

Question Title

* 17. Which disease phenotypes would be most valuable to model or measure? (select all that apply)

Question Title

* 18. Given the ability to test assets in models reflecting diverse patients, in what ways would you want to use the platform? (select all that apply)

Question Title

* 19. How would you prefer to engage with the AMPA platform?

Question Title

* 20. Please provide the name of your organization.

Question Title

* 21. As we validate and test the platform, would you be interested in participating as a beta tester?

Question Title

* 22. Would you be willing to sign a non-binding Letter of Support (LOS) expressing interest in collaborating on developing preclinical models of sporadic ALS (such as AMPA)?

Question Title

Question Title

* 24. Would you be interested in participating in a virtual industry workshop to discuss shared challenges and opportunities in preclinical modeling for sporadic neurodegenerative diseases?

Question Title

* 25. If yes, please provide the contact details for participating in the virtual industry workshop (optional):

Question Title

* 26. Please feel free to provide any additional comments: