Transcript P10288 - OneConference / Dark

The Plan for a World
without T1D
JDRF Translational Development:
Partnering for Success
Turning TypeOne to
What We’ll Cover

JDRF’s Vision for the Future

JDRF’s strategic areas of research

JDRF’s translational strategy

JDRF’s efforts in partnering

Q&A
JDRF’s Vision for the Future
LESS
UNTIL
NONE
LESS
LESS
LESS
LESS
Burden
UNTIL
Invasive
Complexity
Danger
LESS UNTIL NONE
NONE
Therapies
Delivery of Continuous
Therapeutic Improvements
Side-Effects
Complications
Patient Burden
Glycemic Control
Safety
Normal Physiology
Restored
Easier, Safer, Less Worry, Better Life
Cure
Artificial Pancreas
A series of systems that will
automate insulin dosing and
additional hormone delivery
APP Strategy: Iterative Increases in Automation
1 2 3 4 5 6
1
Low Glucose
Suspend
Predictive Low
Glucose
Suspend
Treat-to-Range Treat-to-Target
(w/meal bolus)
Treat-toTarget
(automated)
Treat-to-Target
(multi-hormone,
automated)
Artificial Pancreas
Artificial
Smart Insulin
Pancreas
ASingle
seriesdose
of systems
of insulin
thatthat
will
delivers
automate
insulin
insulin
onlydosing
whereand
and
additional
when
hormone
neededdelivery
Artificial Pancreas
Encapsulation
Smart
Insulin
Smart Insulin
Implantable,
Single dose
betaofcell
insulin
replacement
that
delivers
therapies
insulin
restoring
only where
insulin
and
independence
when
without
needed
the need for
intensive immune suppression
Artificial Pancreas
Encapsulation
Prevention
Smart Insulin
Slow
Implantable,
or stop the
beta
progression
cell replacement
of T1D
before
therapies
insulin
restoring
dependence
insulin
and
independence
ultimately eliminate
without
thethe
possibility
need for
intensive
of everimmune
developing
suppression
T1D
Encapsulation
Restoration
Prevention
Smart Insulin
Artificial Pancreas
Prevention
Slow
Betaorcell
stop
growth
the progression
protected from
of T1D
immune
before insulin
attackdependence
restoring insulin
and
ultimately
independence
eliminate the
without
possibility
ofimmune
ever developing
suppression
T1D
Encapsulation
Near Term
Continuous Therapeutic Improvements
Complications
Diabetic Eye Disease
Artificial Pancreas
Low Glucose Suspend
Predictive Diagnostics
Mid Term
Treat-to-Range
Artificial Pancreas
Encapsulation
Smart Insulin
Treat-to-Target
Implantable Islets
Control
Prevention
Complications
Restoration
Primary: Vaccine
Comprehensive
Prevention Therapy
Prevention
Secondary Prevention
Long Term
Multihormone
New Beta Cell Growth
Protected from Immune Attack
Partnering for Success
Question #1
What is translational research?
a. Research that catalyzes innovative discoveries to
move from bench to the clinic
b. Research which enables scientific discoveries to
mature into potential products
c. Research that progresses through the
developmental pipeline
d. All of the above
Question #2
What is the “Valley of Death”?
a. It’s the location of a JDRF fundraising bike rides
b. It’s the home of a leading T1D research
institution
c. It’s a TV series from which JDRF receives
royalties
d. Slows development of potential cures for T1D
Question #3
What is one reason partnering is essential to JDRF’s
strategy?
a. Partnering is more fun
b. Partnering get you more friends
c. Companies will eventually deliver products to
patients
d. None of the above
The Plan: Work the Pipeline – Translational
Strategy and Partnering for Success
The “Valley of Death” is a Key Part of the Problem
Basic Research
Translational Research
Commercialization
“Valley of Death”
Slows Cures
“The valley of death has gotten
morehas
fatal over the last few years.”
“Thedramatically
valley of death
• Identifying causes
of disease
• Identifying potential
therapeutic targets
Understanding disease
NewsWeek
gotten dramatically
more
fatal over the last few
• Identifying
years.”
therapies
-NewsWeek
• Preclinical
research
Developing Cures
• Human trials
• Drug launches
• Post-launch
studies
Launching Products
JDRF Strategies to Overcome Pipeline Hurdles
1
Enhance Academic and T1D Foundation Collaborations
2
Novel Models for Industry Partnerships
3


Eliminate redundancy and improve efficiencies
Focused on translational research
Engage Strategically Across the Pipeline

Stronger integration of Research and Advocacy efforts and build
additional healthcare assessment capacity
How JDRF collaborates with industry:
Project-based Funding
DELIVERY TO
PATIENTS
JDRF and The Helmsley Charitable Trust:
Sensor Initiative
Project Goals
JDRF-HCT Sensor Initiative aims to
advance CGM accuracy and reliability
towards the next generation artificial
pancreas systems.
• Accurate
• Reliable
• Redundant
• Form factor
• Safe
• Enabling artificial pancreas systems
22
Tandem: Novel Dual-Chamber Infusion Pump
Project Goals
Create a “dual-chamber” pump device that can
simultaneously deliver insulin and other drug therapies.
Dual
Chamber
Infusion
Pump for
insulin and
other
hormones
23
Glucagon in a bi-hormonal pump
Project Goals
Develop a soluble and stable glucagon drug for use in multihormonal artificial pancreas systems
24
How JDRF collaborates with industry: JDRFPharma-Academic Partnering
Joint-research collaboration programs to fund academic research in
partnership with industry.
CENTER FOR
THERAPEUTIC INNOVATION
25
How JDRF collaborates with industry:
Venture Creation, Seed Funding
Advance T1D
breakthroughs across
the valley of death
through a novel,
entrepreneurial
partnership focused
on cures.
Artificial Pancreas
Encapsulation
Smart Insulin
Prevention
Restoration
Complications
Vision
Plan
Capabilities
Your Support