Gene Therapy for Rare Disorders (March 7-10)

Precision will be at this year’s Gene Therapy for Rare Disorders from March 7 – 10^th in Boston. Connect with our team of experts as they lead meaningful presentations and discussions throughout this event providing insights into the clinical development, manufacturing, and commercialization of therapies for rare genetic disorders.

Speaking Engagements
Detailed descriptions for each speaking engagement included below.  

• Precision ADVANCE is the lead sponsor at this year’s Gene Therapy for Rare Disorders conference and will host, “A Patient-centric Approach to the Development of Advanced Therapies” on the morning of Day 1 (Tuesday, March 8^th at 9AM ET) with FARA Spokesperson Kyle Bryant.
• Project Farma is a program partner and will host, “Building a Phase-Appropriate Gene Therapy Manufacturing Facility” with senior leadership from Taysha Gene Therapies on Tuesday, March 8^th at 11:30AM ET.
• Precision for Medicine is a program partner and will have two speaking engagements:
  • Presentation: Nab, Tab and ELISpot – Does it Matter? on Tuesday, March 8^th at 12PM ET (Analytical & Process Development Track)
  • Panel: Thinking Outside of the Box: Clinical Development for Rare Genetic Disorders on Tuesday, March 8^th at 12:30PM ET(Clinical Track)

Connect with our Cell & Gene Therapy Experts

• Visit us at booth (20 & 21) in the exhibition area
• Schedule in-person or virtual meetings by emailing precisionadvance@precisionmedicinegrp.com
• Attend our ADVANCE-sponsored networking reception on Tuesday, March 8th from 6:45 – 7:45PM ET.
• Join Project Farma’s breakfast briefing “Leaders in Manufacturing” on Wednesday, March 9^th from 7 – 7:50AM ET. Register HERE.

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Panel Discussion: A Patient-centric Approach to the Development of Advanced Therapies
Precision ADVANCE | Tuesday, March 8^th 9 AM ET

Ensuring your organization is patient-centric not only delivers more relevant and impactful patient outcomes, but it also can increase speed, efficiency, and productivity in the development advanced medicines. How do we ensure that patients are always top-of-mind during drug development? How has having a patient-focused mindset made a difference in finding cures for rare genetic orders?

• Hear from Kyle Bryant, Spokesperson at FARA (Friedreich’s Ataxia Research Alliance), on his perspective as a patient living with a degenerative neuro-muscular disorder and the impact the gene therapy community is making for patients and their families.
• Learn how a patient-focused mindset can optimized speed-to-market for the development of gene therapies.

Speakers:

• Anshul Mangal: President (Project Farma / Precision ADVANCE)
• Kyle Bryant: Spokesperson at FARA (Friedreich’s Ataxia Research Alliance)
• Phil Cyr: SVP (Precision Value & Health / Precision ADVANCE)

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Panel Discussion: Building a Phase-Appropriate Gene Therapy Manufacturing Facility
Project Farma | Tuesday, March 8^th 11:30 AM ET (Commercial Manufacturing)

This panel discussion will consist of senior leadership from Taysha Gene Therapies and Project Farma to discuss the challenges faced when scaling up a cutting-edge gene therapy facility. Much of the discussion will revolve around mitigation of risk while building an innovative phase-appropriate gene therapy facility under accelerated timelines. The panel features manufacturing leaders who have pioneered gene therapy facility builds including build strategy, facility scale-up and operational readiness.

Attendees will learn about:

• The site selection process including a “make vs. buy” business case analysis
• Utilizing modular and single-use technologies
• Ensuring proper workstreams, phased implementation, and commissioning and qualification strategies
• Facility infrastructure, quality, and automation systems for gene therapy facilities

Speakers:

• [Moderator] Anshul Mangal: President (Project Farma / Precision ADVANCE)
• Fred Porter: CTO (Taysha Gene Therapies)
• Greg Gara: SVP of Manufacturing (Taysha Gene Therapies)
• Tony Khoury: EVP (Project Farma / Precision ADVANCE)
• Joe Neroni: Director (Project Farma / Precision ADVANCE)

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Presentation: NAb, TAb, and ELISpot – Does it Matter?
Precision for Medicine | Tuesday, March 8^th 12 PM ET (Analytical & Process Development)

Attendees will learn about:

• Rationale for each assay
• Correlation between assay types
• Regulatory considerations 

Presenter:

• Lisa Hoover: Associate Director, Cellular Assays (Precision for Medicine / Precision ADVANCE)

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Panel Discussion: Thinking Outside of the Box: Clinical Development for Rare Genetic Disorders
Precision for Medicine | Tuesday, March 8^th 12:30 PM ET (Clinical)

Attendees will learn about:

• Identifying challenges in clinical development for rare genetic disorders
• Understanding the patient journey, patient identification and recruitment
• Finding creative solutions for clinical development
• How natural history studies inform decisions in clinical development

Speakers:

• Teresa Pokladowski: Director, Strategic Development (Precision for Medicine / Precision ADVANCE)
• Courtney Wells: SVP, Clinical Operations & Clinical Affairs (Jaguar Gene Therapy)