N1C Workgroups bring together individuals from across the ecosystem to collaboratively assess and advance key areas of individualized therapy development. Each workgroup focuses on a specific domain, creating space for participants to share experiences, examine current approaches, and contribute to the development of practical guidance and resources.
Patient Identification
Developing consensus-driven frameworks and tools to identify patients who are appropriate candidates for individualized genetic therapies. LEARN MORE
Preclinical Design
Advancing best practices for designing, testing, and evaluating individualized genetic therapies through collaborative preclinical research. LEARN MORE
Manufacturing
Sharing practical manufacturing and CMC guidance to help researchers navigate GMP requirements and accelerate therapy development. LEARN MORE
Institutional Implementation
Building shared operational frameworks, resources, and best practices to help institutions implement N-of-1 therapy programs. LEARN MORE
Clinical Outcomes
Developing guidance for selecting meaningful clinical outcome measures that evaluate safety, efficacy, and patient benefit in N-of-1 trials. LEARN MORE
Data Coordination
Connecting the community with data initiatives to improve collaboration, interoperability, and the development of shared data resources. LEARN MORE
Access & Funding
Identifying funding pathways, strategic partners, and evidence needed to improve access to and long-term sustainability of individualized therapies. LEARN MORE
Regulatory
Synthesizing regulatory experience and practical guidance to help accelerate approvals for individualized genetic therapies. LEARN MORE
Patient Identification
Developing consensus-driven frameworks and tools to identify patients who are appropriate candidates for individualized genetic therapies. LEARN MORE
Preclinical Design
Advancing best practices for designing, testing, and evaluating individualized genetic therapies through collaborative preclinical research. LEARN MORE
Manufacturing
Sharing practical manufacturing and CMC guidance to help researchers navigate GMP requirements and accelerate therapy development. LEARN MORE
Institutional Implementation
Building shared operational frameworks, resources, and best practices to help institutions implement N-of-1 therapy programs. LEARN MORE
Clinical Outcomes
Developing guidance for selecting meaningful clinical outcome measures that evaluate safety, efficacy, and patient benefit in N-of-1 trials. LEARN MORE
Data Coordination
Connecting the community with data initiatives to improve collaboration, interoperability, and the development of shared data resources. LEARN MORE
Access & Funding
Identifying funding pathways, strategic partners, and evidence needed to improve access to and long-term sustainability of individualized therapies. LEARN MORE
Regulatory
Synthesizing regulatory experience and practical guidance to help accelerate approvals for individualized genetic therapies. LEARN MORE
What Participation Looks Like
Workgroups are collaborative, discussion-driven environments where individuals:
-
- Share real-world experience and expertise
- Contribute to discussions around current practices and challenges
- Help inform the development of shared guidance, tools, and resources
Contributions are recognized at the individual level (not companies), with participants acknowledged in any resulting outputs.
Why It Matters
Individualized therapy development remains complex and fragmented. Workgroups help bring clarity by:
- Surfacing what is currently working
- Identifying gaps and challenges
- Supporting more consistent and informed approaches across the field
Who Should Join
Workgroups are designed for individuals who are:
- Actively working in areas related to individualized or ultra-rare disease therapeutics
- Interested in contributing their expertise to collaborative discussions
- Open to sharing experiences and learning from others across disciplines
This includes biotechnology and pharmaceutical companies, as well as organizations supporting research, development, and delivery of individualized therapies.
Get Involved
If you are interested in joining a workgroup, please click the button below to fill out an inquiry form. It will be reviewed by our team and you will receive a response within 3 business days.
What Participation Looks Like
Workgroups are collaborative, discussion-driven environments where individuals:
-
- Share real-world experience and expertise
- Contribute to discussions around current practices and challenges
- Help inform the development of shared guidance, tools, and resources
Contributions are recognized at the individual level (not companies), with participants acknowledged in any resulting outputs.
Why It Matters
Individualized therapy development remains complex and fragmented. Workgroups help bring clarity by:
- Surfacing what is currently working
- Identifying gaps and challenges
- Supporting more consistent and informed approaches across the field
Who Should Join
Workgroups are designed for individuals who are:
- Actively working in areas related to individualized or ultra-rare disease therapeutics
- Interested in contributing their expertise to collaborative discussions
- Open to sharing experiences and learning from others across disciplines
This includes biotechnology and pharmaceutical companies, as well as organizations supporting research, development, and delivery of individualized therapies.
Get Involved
If you are interested in joining a workgroup, please click the button below to fill out an inquiry form. It will be reviewed by our team and you will receive a response within 3 business days.