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Home  >  Conferences  >  Inborn Errors of Metabolism Drug Development Summit 2020

Inborn Errors of Metabolism Drug Development Summit 2020

Mar 10 - 12, 2020   |   Boston, Massachusetts  United States of America
Organized by : Hanson Wade
Specialties : Pharmacy and Medicine   |   Genetics
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Conference Brochure

Inborn Errors of Metabolism Drug Development Summit is organized by Hanson Wade and will be held from Mar 10 - 12, 2020 at Courtyard by Marriott Boston Downtown/North Station, Boston, Massachusetts, United States of America.

Main Conference - March 11 - 12, 2020
Pre Conference Workshops - Tuesday, March 10

Description:
As the field of inherited metabolic disease looks to assess the viability of pioneering gene therapy modalities, drug development within existing pharmacology continues as second generation enzyme replacement and small molecule therapies progress through the clinic. Regardless of the therapeutic modality you aim to build, all drug developers are working to fulfil the huge unmet medical need that exists for inherited metabolic disorders. In order to accelerate drug development and address this unmet medical need, a set of unique and complex challenges associated with rare metabolic diseases must be overcome. If these challenges can be addressed drug developers can be confident that their therapeutic modalities will achieve their full commercial potential.

• Dissect the IEM therapeutic landscape to discuss the future of gene therapy modalities, ERT’s, small molecule and substrate reduction therapies treating rare metabolic disorders and refresh your understanding of IEM drug development progress
• Explore novel and existing delivery pathways in order to more effectively bring your therapeutic to the site of action
• Address patient access and recruitment in an increasingly competitive landscape to more effectively source the right patient for the right trial
• Identify robust clinical endpoints and biomarkers to better understand disease progression and therapeutic outcome
• Harness the power of natural history research and rethink your approach to clinical trial design to confidently produce meaningful clinical endpoints
• Analyse current regulatory requirements to better navigate the regulatory landscape and more confidently progress your drug development pipeline
• Address the approval, investment and reimbursement challenges of IEM drug development to accelerate your therapeutics from the clinic to commercialization
• Highlight unmet medical needs for ultra-rare disease to drive forward investment and accelerate drug development

Pre Conference Workshops:
Workshop A - Patient Access & Recruitment for Rare & Ultra-Rare Metabolic Diseases
Time: 8:00 - 11:00
Description:
As the rate of drug development for inherited metabolic diseases continue to increase, access to an already limited population of patients for clinical trials becomes progressively competitive.

This workshop will look to tackle the fundamental challenges that are associated with developing drugs for rare diseases with inherently low patient population numbers. Join this workshop to explore how industry and patient advocacy collaborations can work to maximize patient recruitment and retention. Further your understanding of patient eligibility for clinical trials to more confidently match the right patient to the right trail.

Workshop B - Identifying Meaningful Clinical Endpoints in Rare Metabolic Disease Trials
Time: 11:30 - 2:30
Description:
This workshop will look to explore how meaningful clinical endpoints can more confidently be identified in rare metabolic disorder trials. Evaluate current clinical trial design and explore how a more ‘patient centric’ focus can work to better identify and validate meaningful endpoints for the patient. Furthermore, discuss how therapeutic testing protocol can be better designed in order to develop a standardized methodology that more effectively tracks diseases progression and therapeutic outcomes.

Workshop C - Natural History Studies: Looking Backwards to Move Forward
Time: 3:00 - 6:00
Description:
This session will look to highlight how using natural history studies and acquired learnings from drug development progress of the field can inform future decision making.

Harness the power of natural history research and rethink your approach to clinical
trial design to more confidently produce meaningful clinical endpoints.

Explore how collaborative approaches to natural history studies can reduce study
repetition and the burden placed on patients.

Additional details will be posted as soon as information is available.

View All Speaking

Sonia Poli
Sonia Poli

Biochemistry and Molecular Genetics

Arthur Taveras
Arthur Taveras

Biochemistry and Molecular Genetics

Lex M. Cowsert
Lex M. Cowsert

Immunology and Microbiology

Conference Details

Exhibit Details : View Exhibit
Session url : Click here

Registration Desk

Contact No. : (+1) 617 455 4188

Email:
info@hansonwade.com
register@hansonwade.com

Registration

Registration Type Last Date Price
Academic Pricing: Conference Plus 3 Workshops - Final Fee 09 Mar,2020 USD 3079.00
Academic Pricing: Conference Plus 2 Workshops - Final Fee 09 Mar,2020 USD 2729.00
Academic Pricing: Conference Plus 1 Workshop - Final Fee 09 Mar,2020 USD 2379.00
Academic Pricing: Conference Only - Final Fee 09 Mar,2020 USD 2029.00
Academic Pricing: Workshop Only - Final Fee 09 Mar,2020 USD 599.00
Standard Pricing: Conference Plus 3 Workshops - Final Fee 09 Mar,2020 USD 4396.00
Standard Pricing: Conference Plus 2 Workshops - Final Fee 09 Mar,2020 USD 3897.00
Standard Pricing: Conference Plus 1 Workshop - Final Fee 09 Mar,2020 USD 3398.00
Standard Pricing: Conference Only - Final Fee 09 Mar,2020 USD 2899.00
Standard Pricing: Workshop Only - Final Fee 09 Mar,2020 USD 599.00
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Venue Address

Courtyard by Marriott Boston Downtown/North Station
107 Beverly Street
Boston, Massachusetts, United States of America

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Hotels Near Venue

Freepoint Hotel Cambridge, Tapestry Collection by Hilton
4.55 miles from venue
Price : USD 259.00 Per Night
The Charles Hotel in Harvard Square
3.22 miles from venue
Price : USD 649.00 Per Night
Sonder — Pierce Boston
2.65 miles from venue
Price : USD 0.00 Per Night
Hyatt Regency Cambridge, Overlooking Boston
2.52 miles from venue
Price : USD 409.00 Per Night
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Session Details

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Workshop Details

Title Start Time End Time Workshop Fee
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Exhibit Details

Start Date End Date Exhibit Fee
01 Jan 12:00 AM 01 Jan 12:00 AM ALL 2
01 Jan 12:00 AM 01 Jan 12:00 AM 0
01 Jan 12:00 AM 01 Jan 12:00 AM ALL 2
01 Jan 12:00 AM 01 Jan 12:00 AM ALL 2
01 Jan 12:00 AM 01 Jan 12:00 AM USD 4
01 Jan 12:00 AM 01 Jan 12:00 AM USD 4