Shire Partnership Opportunity to Develop Rare Disease Therapeutics
Shire Human Genetic Therapies (Shire HGT), the Technology & Innovation Development Office
(TIDO) and the Manton Center for Orphan Disease Research at Boston Children’s Hospital are
pleased to announce a new resource for funding and supporting therapeutic development for rare
diseases. Shire HGT’s program is specifically designed for Boston Children’s investigators.
Program goal:
Collaborative development of therapeutics for rare diseases.
Scope:
Shire HGT will support development of therapeutics for rare diseases, excluding rare cancers.
Focus will be on programs that can deliver a therapeutic candidate molecule in less than 3
years.
Projects will be selected by Shire HGT based on the following criteria:
 Alignment with Shire HGT strategy to treat rare diseases
 Clearly defined proposals that are testable in <3 year timeframe
 Acceptable knowledge of disease biology/mechanism of action
 Target modulation as expected by mechanism of action
 Animal model(s) or other appropriate experimental system(s) indicates reasonable
likelihood of translation to humans
 Outline of biomarker strategy facilitates translation to the clinic
Each project will be undertaken by a joint team, with members from both the academic group
and Shire HGT working towards agreed common goals. Shire HGT will contribute experienced
drug development team members to every partnership to meet the specific needs of each project.
Projects will be scoped through early clinical studies (phase I).
Complementary Shire capabilities:
Specific technologies available for utilization:
1. Protein therapeutic development expertise, e.g. in enzyme replacement therapies.
2. Delivery of large molecules to the CNS.
3. Oligonucleotide-based therapeutics.
4. Antibody therapeutics.
5. Gene therapy.
6. Small molecule therapeutics.
7. Computational biology.
General expertise available through collaboration: see page 3.
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Application:
Application process and timeline:
1. Investigators will submit 2 page non-confidential proposals to the joint steering
committee (JSC) through TIDO.
2. The JSC will select preliminary projects.
3. The JSC will invite investigators whose proposals were selected to submit a 5-6 page
confidential proposal detailing the project and its goals.
4. Investigators present proposals to the JSC, after which Shire HGT makes final selections.
Important dates:
1. January 21th 2013: Pre-proposals due
2. Early February: Notification of invitation to submit a full proposal
3. Mid-March: Full proposals due
4. Early April: Presentation to steering committee
5. Late April: Award notification
6. May-June: drafting of project plans and project launch
Funding level:
Mutually-approved budget commensurate with project needs. Award funding includes salaries,
supplies, and indirect costs.
Benefits to investigators:
 A therapeutic development partner from idea to product
 The opportunity to drive a project from lab to clinic to meet the medical needs in
Children’s patient population
 Partnership with a team from Shire with complementary drug development expertise
 Access to a broad array of people, tools, techniques and knowledge within the Shire
scientific community
 Commitment to joint publications and generation of new research tools
 A financial share in the future success of a product
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Shire HGT: Selected Capabilities
Our overarching purpose at Shire is to enable people with life-altering conditions to lead better
lives. Shire Human Genetic Therapies (HGT) brings to the table the proven ability to transition a
concept from benchtop to the clinic and the market, ultimately delivering therapeutic products in
the rare space to the patients with high unmet need. Numerous areas of expertise are critical for
successful advancement of a candidate through the complex drug development process. For this
purpose, HGT’s proficiency in biomolecular research, nonclinical and clinical development will
be briefly highlighted.
Biomolecular Research provides proficiency in molecular biology, cell line development in a
variety of hosts, protein purification, protein analytics, protein crystallography, assay
development and phage display library screening. Through high quality instrumentation and
expertise to examine protein-protein interactions, enzyme kinetics, protein stability, and in vitro
functional assays, the team has the ability to provide drug products, assay reagents, functional
assays, critical information on potential drug targets, and evaluation of samples from natural
sources.
Nonclinical Development consists of Toxicology, Clinical Pharmacology and
Pharmacokinetics, and is responsible for all nonclinical in vivo studies from the early research
phase throughout the clinical development and post-market phases. In support of research
projects, the group evaluates the biodistribution, initial pharmacokinetics and in vivo proof-ofconcept (POC) studies efficacy for each therapeutic candidate, and provides strategic program
support through the targeted publication of results. For programs entering development, during
development and post-approval, the group conducts PK-PD studies to define clinical safe starting
dose and efficacious dose and regimen, and to support safety aspects of the clinical development
program. This work typically involves expanding upon initial preclinical research proof-ofconcept data in Pharmacology, Pharmacokinetics, ADME, Safety Pharmacology, and
Toxicology (including Acute, Subchronic, Chronic, Reproductive and Juvenile Toxicology). A
key part of the team’s support for programs at all stages of development is contributing critical
data and interpretation to worldwide Regulatory filings and Regulatory Agency interactions,
including Pre-IND/FIH/Registration/Amended Filings (e.g. IND, NDA/BLA, MAA, eCTD, 2nd
Gen Products).
Clinical Development - Shire HGT brings Clinical Capabilities in Rare Drug Development that
have successfully moved several compounds from internal research to global registration. The
core clinical strengths begin with understanding the science of the compound, development of
appropriate epidemiology for rare diseases and expertise in the regulatory requirements needed
for the development of novel registerable endpoints. Shire HGT has repeatedly demonstrated the
ability to move from fluency in the basic biology of the rare disease to development of a detailed
knowledge of the disease natural history and response to therapies. This experience highlights
the proven track record of moving from available laboratory and clinical measures in rare
diseases without a defined regulatory path into the delivery of successful clinical trials utilizing
clinically meaningful endpoints.
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