Digital Conference Agenda

9:00 am - 4:00 pm EDT      |      6:00 am - 1:00 pm PST

* Please note that the following agenda timings are Eastern Time.  

For Pacific Times, please download the full event guide here

8:15 am Online Registration and Coffee Networking Session

8:15 am Online Registration and Coffee Networking Session

8:15 am Online Registration and Coffee Networking Session

8:15 am Online Registration and Coffee Networking Session

8:15 am Online Registration and Coffee Networking Session

Synopsis

Kick start your conference with a virtual networking experience. Grab your morning cup of coffee, sit back and join the conversation

8:15 am Online Registration and Coffee Networking Session

8:15 am Online Registration and Coffee Networking Session

8:15 am Online Registration and Coffee Networking Session

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

Delivering Your Therapeutic to the Area of Action

Delivering Your Therapeutic to the Area of Action

Delivering Your Therapeutic to the Area of Action

Delivering Your Therapeutic to the Area of Action

Delivering Your Therapeutic to the Area of Action

Delivering Your Therapeutic to the Area of Action

Delivering Your Therapeutic to the Area of Action

Delivering Your Therapeutic to the Area of Action

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

Synopsis

• Exploiting natural tropism of lentivirus vectors
• Permanent genetic modification suitable for infants
• New strategies to improve hepatocyte transduction

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

Synopsis

• Discuss design considerations for antibody-guided enzymes
• Share proof-of-concept data on the use of antibody-guided enzymes for the treatment of lysosomal diseases
• Discuss delivery methods for antibody-guided enzymes, including gene therapy

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases

10:00 am Live Q&A Session

Synopsis

  • Have the speakers answer your questions

10:00 am Live Q&A Session

10:00 am Live Q&A Session

10:00 am Live Q&A Session

10:00 am Live Q&A Session

10:00 am Live Q&A Session

10:00 am Live Q&A Session

10:00 am Live Q&A Session

10:30 am Morning Refreshments & Virtual Speed Networking

10:30 am Morning Refreshments & Virtual Speed Networking

10:30 am Morning Refreshments & Virtual Speed Networking

10:30 am Morning Refreshments & Virtual Speed Networking

10:30 am Morning Refreshments & Virtual Speed Networking

10:30 am Morning Refreshments & Virtual Speed Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

10:30 am Morning Refreshments & Virtual Speed Networking

10:30 am Morning Refreshments & Virtual Speed Networking

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

Synopsis

• The trojan horse platform of antibody-enzyme fusion proteins to transport therapeutics across the blood-brain-barrier
• Discovery, validation and preclinical development of Valanafusp alpha, aka AGT-181
• Phase II clinical proof of concept of Valanafusp alpha and its effect on pediatric patients with severe untransplanted MPS I
• Regulatory pathway and clinical endpoints discussed with FDA and EMA for a pivotal trial with Valanafusp alpha

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

11:30 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

Synopsis

• mRNA and lipid nanoparticles science and technology
• Preclinical and non-clinical studies in inherited metabolic disorders
• Future and applications of mRNA

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

12:00 pm MRNA Therapy for Inherited Metabolic Disorders

12:20 pm Live Q&A Session

12:20 pm Live Q&A Session

12:20 pm Live Q&A Session

12:20 pm Live Q&A Session

12:20 pm Live Q&A Session

12:20 pm Live Q&A Session

Synopsis

• Have the speakers answer your questions

12:20 pm Live Q&A Session

12:20 pm Live Q&A Session

12:50 pm Lunch

12:50 pm Lunch

12:50 pm Lunch

12:50 pm Lunch

12:50 pm Lunch

12:50 pm Lunch

12:50 pm Lunch

12:50 pm Lunch

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

Synopsis

• Development in X-ALD and Friedreich ataxia
• Translational models
• Clinical endpoints and biomarkers: challenges and opportunities

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

1:50 pm Development of Leriglitazone in Rare Neurodegenerative Diseases

  • Sonia Poli Development & Strategy Advisor, Minoryx

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

Synopsis

• Developing a better understanding of transcriptional control and harnessing this for disease modulation
• Which diseases can this approach be applied to?

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

2:20 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

2:50 pm Live Q&A Session

2:50 pm Live Q&A Session

2:50 pm Live Q&A Session

2:50 pm Live Q&A Session

2:50 pm Live Q&A Session

2:50 pm Live Q&A Session

Synopsis

• Have the speakers answer your questions

2:50 pm Live Q&A Session

2:50 pm Live Q&A Session

3:20 pm Business Development in Rare Disease

Synopsis

• Evaluating Orphan Assets
• Evaluating drug candidates for partnering & investment
• Finding rare disease assets to match development goals
• Assessing asset suitability for deal-making

3:20 pm Business Development in Rare Disease

3:20 pm Business Development in Rare Disease

3:20 pm Business Development in Rare Disease

3:20 pm Business Development in Rare Disease

3:20 pm Business Development in Rare Disease

3:20 pm Business Development in Rare Disease

3:20 pm Business Development in Rare Disease

3:40 pm Live Q&A Session

3:40 pm Live Q&A Session

3:40 pm Live Q&A Session

3:40 pm Live Q&A Session

3:40 pm Live Q&A Session

3:40 pm Live Q&A Session

Synopsis

• Have the speakers answer your questions

3:40 pm Live Q&A Session

3:40 pm Live Q&A Session

3:50 pm Chair’s Closing Remarks

3:50 pm Chair’s Closing Remarks

3:50 pm Chair’s Closing Remarks

3:50 pm Chair’s Closing Remarks

3:50 pm Chair’s Closing Remarks

3:50 pm Chair’s Closing Remarks

3:50 pm Chair’s Closing Remarks

3:50 pm Chair’s Closing Remarks

4:00 pm End of Day One

4:00 pm End of Day One

4:00 pm End of Day One

4:00 pm End of Day One

4:00 pm End of Day One

4:00 pm End of Day One

4:00 pm End of Day One

4:00 pm End of Day One