KIF1A.ORG has a unique and urgent approach in partnering with pharmaceutical and biotech companies to discover treatment for our children. Working closely with the KIF1A research and medical team, we make it easier for a company to execute strategy and create programs focused on rapid development of treatment for KAND.

As a patient-led research organization, we have a responsibility to bring efficient and organized tools to the table. These tools are freely available to the scientific community, including biotech and pharma.

Efficient Collaboration

These tools support every function of development — from pre-clinical discovery through clinical trial readiness and regulatory approval. Our organized resources are scaled to support any therapeutic program. Offering these tools supports your process and makes KIF1A.ORG the ideal partner in efficiently addressing our significant unmet therapeutic need.

This KIF1A toolkit crosses off industry line items in a validated, scientific approach. These tasks typically require significant time and resources, and are often the reason companies choose not to invest in a rare disease program. Not with KIF1A. We make it faster and easier for any pharma or biotech company that sees an opportunity to partner with KIF1A.ORG.

Pre-Clinical Capabilities

  • Detailed natural history and genotype/phenotype studies
  • Detailed and re-contactable patient registry
  • List of pathogenic mutations
  • Animal models and iPSC lines of mutations with diverse phenotypes
  • Rapid response and open dialogue with clinical research team
  • Rapid response and open dialogue with families and foundation

Clinical Trial Readiness

  • Detailed re-contactable patient registry to support rapid enrollment
  • Global map of patient locations to support trial site feasibility and selection
  • Center of excellence with trial experience and capabilities ready to activate
  • Families willing to support trial design and protocol development
  • Meaningful clinical endpoints and outcome measures

Advocacy for Regulatory Approval

  • Educated families to advocate with company for approval
  • Detailed description of impactful and meaningful outcome measures
  • Structured PFDD model prepared with clear message
  • Impactful videos of families illustrating unmet needs and meaningful change
  • Physicians ready to advocate for approval
  • Data on frequency and global need for treatment

Advocacy for Market Access

  • Detailed patient journey stories freely accessible
  • Family advisory board to support formulation decisions
  • Detailed understanding of diagnostic pathway
  • Rapid response and open dialogue with clinicians
  • Rapid response and open dialogue with patient families

 

KIF1A Capabilities

Currently, we have full pre-clinical capabilities and are actively looking to partner with pharmaceutical and biotech companies to develop treatment for our kids. We have an enormous unmet therapeutic need, and time is our enemy with a progressive neurodegenerative disorder. Because of this, we’re prepared to play an active role in your process by freely providing all of these tools to industry partners.

KIF1A.ORG is not interested in intellectual property, patents or profit. We’re a group of patients, advocates, caregivers, family members and scientists willing to do whatever it takes to give our kids with KAND a meaningful and happy quality of life.

Applicable to Any Rare Disease

Tools for Development is an efficient model that any organized and driven rare disease foundation can employ. We’re more than happy to share this process with any rare disease community. Our hope is to help find treatment for every child living with a rare genetic disease.