Advanced Therapies Week is the destination for anyone developers, investors or those interested in cell and gene therapies and tissue engineered products. Throughout the week, the organizers be host a variety of networking events, focused roundtables and content sessions designed to help biotechs advance along their commercialization journey.

Speaker: Sheila Mikhail, CEO & Co-Founder, AskBio
Presentation & Q&A Panel

AskBio’s Sheila Ann Mikhail joined Matter’s Tales from the Trenches to share her entrepreneurial journey, her perseverance and vision for AskBio and lessons learned throughout her career.

AskBio’s Graham Whyteside, PhD, Director, Platform R&D, AskBio Europe, spoke at the Synthetic Biology-Based Therapeutic Summit on Advancing Synthetic Promoter Design.

What does it take to shatter the glass ceiling in biopharma R&D?

Calls to diversify the biopharma industry have grown louder over the last year, and in response, more companies have made laudable efforts to address the issue. AskBio’s Sheila Mikhail sat down with other leaders from across the biopharma industry to discuss breaking the glass ceiling, the limitations of existing gender diversity programs, and what comes next.

Pre-Recorded Company Presentation by Sheila Mikhail, CEO & Co-Founder, AskBio

Meeting web site: Cell & Gene Meeting on the Mesa

More than 200 NCBIO members and invited guests come together to enjoy a program of speakers and panels addressing current topics of importance to the industry.

Keynote speaker Jude Samulksi, Ph.D., President and CSO of AskBio will share about his start in North Carolina, current issues and what comes next in the biotechnology space.

Company Presentation (Fireside Chat Format)

Speaker: Sheila Mikhail, Chief Executive Officer and Co-Founder, AskBio

Dual Dialogue – Anatomy of an Acquisition: Bayer and AskBio

• Overview of the structure of the acquisition, goals, outcomes
• How the deal came about and COVID-19’s impact on the transaction
• Bayer’s unique operating model to ensure to successful partnerships within its Cell and Gene Therapy Platform
• Advancing AAV manufacturing post-acquisition

Speakers:

Sheila Mikhail – CEO & Co-Founder, AskBio

Marianne De Backer, MBA, PhD – EVP, Head of Strategy, BD&L, Bayer

The World Orphan Drug Congress has become the industry defining meeting on Advanced Therapies for rare diseases, featuring discussions on clinical development, manufacturing and commercialization of cell and gene therapies, as well the annual gathering for artificial intelligence and digital health solutions transforming the rare disease industry.

Keynote Panel: ‘Treating Rare Diseases: Future of Innovation and Development for Cell and Gene Therapies

Thursday, August 26, 2021 9:40 a.m. – 10:25 a.m. ET

Additional information: World Orphan Drug Congress USA 2021 – NORD (National Organization for Rare Disorders) (rarediseases.org)

The 2021 STAT Breakthrough Science Summit will take you inside these innovations, examining how they’re being developed, adopted, and paid for. We’ll also take a look at the breakthroughs that haven’t yet hit the market, but have the potential to redefine health and medicine in the years to come.

Session: The Future of Gene Therapy
Katherine High, M.D., president, therapeutics, AskBioBobby Gaspar, M.D., Ph.D. chief executive officer, Orchard TherapeuticsSekar Kathiresan, M.D., co-founder and chief executive officer, Verve TherapeuticsAdam Feuerstein, senior writer, biotech, STAT (moderator)

Kathy High led the development of the first gene therapy. Now she, along with several other top executives, will guide us through what’s next.

Tuesday, July 13, 2021

1:40 p.m. ET

For 2021, the BIO International Convention will be held virtually as BIO Digital. BIO Digital will feature sessions on key sessions, including a discussion on gene therapy, including an industry panel on new approaches and challenges to developing and delivering genetic medicines.

Session: Realizing the Promise of Gene Therapy: Current Challenges and Future Opportunities
AskBio Presenter: Katherine High, MD, President, Therapeutics
Date/Time: Wednesday, June 16, 2021, 11:05 – 11:50 a.m. (EDT)

The first industry-dedicated Gene Therapy for Muscular Disorders Summit will focus on overcoming the translational and clinical challenges of developing genetic therapies targeting muscular disorders, including Pompe Disease and Limb-Girdle Muscular Dystrophy (LGMD). AskBio presentations include:

Session: Optimizing Gene Therapy Delivery with Next Generation Capsids & Muscle Specific Promoters
Presentation Title: Optimizing the Selectivity of AAV for Target Muscle Cells Using Promoters
Presenter: Juan Iglesias, Director, Product R&D, AskBio
Date/Time: Wednesday, May 26, 2021 11:00 AM EST

Session: Harnessing the Value of Preclinical Model Challenges to Effectively Determine Dose
Panel Title: Q&A Panel Discussion – Overcoming Translational Challenges
AskBio Participant: Michael O’Callaghan, Senior Vice President, Translational Medicine, AskBio
Date/Time: Thursday, May 27, 2021 1:40 PM EST

The East Coast CNS Therapeutics Xchange 2021 brings together executives from pharma and biotech to address and find solutions to the key issues faced in developing CNS therapies. Discussion topics will cover Novel Targets, Preclinical, Clinical Studies, Advanced Therapies and Drug Delivery.

AskBio’s Nathalie Cartier-Lacave will present during the following session:

Session Title: Gene Therapies – How Close Are We to Curing CNS Diseases
Speaker: Nathalie Cartier-Lacave, CNS Gene Therapy Lead, AskBio
Date/Time: May 24, 2021, 9:10 a.m. – 10:10 a.m. ET

For more information about the CNS Therapeutics Xchange, please visit: CNS Therapeutics Xchange | East Coast 2021 – hubXchange (hub-xchange.com)

The Advanced Therapies Congress & Expo 2021 is dedicated to commercialization of cell and gene therapies and regenerative medicines. AskBio will participate in the Congress as part of the following panel sessions:

Keynote Panel Discussion: The Next Generation of Gene Therapy
AskBio Panelist: David Venables, President, AskBio Europe
Date/Time: Thursday, May 20, 2021 13:00 – 14:00 BST

Roundtable: Business Development Strategies
AskBio Panelist: David Venables, President, AskBio Europe
Date/Time: Friday, May 21, 2021 14:30 – 15:30 BST

Additional information regarding the Congress and panels is available here: Research. Develop. Collaborate. Commercialise | Advanced Therapies | 19 – 21 May 2021 (terrapinn.com)

We’re looking forward to the ASGCT 2021 Virtual Meeting being held held May 11-14, 2021. We have a number of presentations and sessions scheduled:

Join us at Gene Therapy Europe, an online congress from May 5-6, 2021 that will feature in-depth discussions on advanced analytical strategies and next-gen technologies to progress gene therapy development from discovery to clinical stages.

Presentations

May 5
8:50-9:10 BST
Advanced AAV Gene Therapy For Neurological Disorders
Ken Macnamara, PhD, AskBio Europe Chief Operating Officer

May 6
16:40-17:00 BST
[Title to come]
David Favre, DMV, VP Translational Medicine

Join AskBio at the 2021 Meeting on the Med for our on-demand sessions. The conference will be held April 6-9, 2021.

Company Presentation

Viewable on demand starting Tuesday, April 6, 2021
David Venables, PhD, President, AskBio Europe

Large Cap Cell and Gene Therapy Strategies

Viewable on demand starting Wednesday, April 7, 2021
Chair: Usman “Oz” Azam, MD, President and CEO, Tmunity Therapeutics
Speakers
Katherine High, MD, President of Therapeutics, AskBio
Bastiano Sanna, PhD, EVP, Chief of Cell and Genetic Therapies and VCGT Site Head, Vertex Pharmaceuticals
Jacob Sten Petersen, DMSc, Corporate VP and Head of Stem Cell R&D, Novo Nordisk
Krishnan Viswanadhan, PharmD, SVP, Global Cell Therapy Franchise Lead, Bristol-Myers Squibb

We’re presenting at the BioProcess International U.S. West conference, which will be held as a virtual event from March 29-31, 2021.

Presentation

Andy Holt – EVP, Commercial Development, AskBio
Platform Development in AAV Manufacturing: Cost & Timeline Impact on Clinical Development
Track 6: Cell & Gene Therapies
Tuesday, March 30
1:15-1:35pm PDT

Founders Discussion

Viral Vector Manufacturing in RTP
Tuesday, March 23, 2021
2:00-3:20pm ET

Josh Grieger, AskBio Chief Technology Officer
Paul Garofolo, Locus Biosciences CEO and co-founder
Ed Field, BioLabs NC President
Kim Nguyen, Precision Biosciences VP CMC
Benjamin Yerxa, Foundation Fighting Blindness CEO

To learn more about CED Venture Connect or register, visit https://cednc.org/venture-connect-2021/.

Join us for BIO Europe Spring, which will be held as a virtual conference from March 22-25, 2021.

On-demand panel discussion

Patient-Centered Drug Development in an Age of Cell and Gene Therapies
Sheila Mikhail, AskBio CEO and co-founder
Rodolphe Clerval, Horama CEO
Craig Martin, Global Genes Board member & Chair, Corporate Alliance (panel moderator)

Join Dr. Krys Bankiewicz for the Emerging Gene Therapy Technology for Neurodegenerative Diseases workshop at the 9th Neurodegenerative Drug Development Summit on February 23, 2021.

Workshop

Emerging Gene Delivery Technology for Neurodegenerative Diseases

Krystof Bankiewicz, MD, PhD, President & CEO, Brain Neurotherapy Bio, CEO, Columbus Children’s Foundation
Tuesday, Feb. 23
1:30-3:30pm ET

Using Parkinson’s, multiple system atrophy and Huntington’s as clinical case studies, this session looks to address the following key topics:

• What novel devices are advancing for gene therapy delivery specifically for neurodegenerative diseases?
• What improvements have there been in the last year?
• What common challenges are we yet to have overcome?
• Considering the route of administration and patient population size, to what extent is gene therapy a viable option for Alzheimer’s Disease and Parkinson’s Disease?
• Advances and progress in developing gene therapies for the rare and ultra-rare neurodegenerative disorders

Join us at the 4th Annual Gene Therapy for Rare Disorders conference, which will be held as a virtual event from February 22-25, 2021. AskBio’s Dr. David Favre will present “Immunotherapeutic Interventions” on Feb. 25 at 1:30pm ET.

Presentation

David Favre, PhD, VP Translational Medicine, AskBio
Immunotherapeutic Interventions
Thursday, Feb. 25 (Discussion Day)
1:30pm ET

Details:

• Pre-existing vs. naïve anti-AAV immunity require different targeted interventions
• B-cell and antibody depletion are key for pre-existing immunity but may not be sufficient
• In naïve subjects: conditioning regimen and B/T -cell mediated immune tolerance induction
• Advanced immune monitoring strategies and experimental medicine trials are key for progressing immunotherapeutic interventions in patient populations

AskBio is an exhibitor at WORLDSymposium 2021, which will be held as a virtual event from February 8-12, 2021. We invite registered attendees to stop by, meet our team, and learn more about our gene therapy technology and clinical programs.

Join gene therapy expert Nathalie Cartier-Lacave for the Advanced Therapies panel at the RE(ACT) Congress & IRDiRC Conference, which will be held as a virtual meeting from January 13-15, 2021.

PANEL

Thursday, January 14, 2020
Session E, 15:30 to 17:00
Advanced therapies: gene editing, cell therapy”

• Joseph Scheeren, USA Challenges and considerations in the regulation of gene and cell therapies”
• Alessandro Aiuti, IT “Hematopoietic stem cell gene therapy for monogenic diseases: from experimental studies to approved drugs”
• Hans-Dieter Volk, DE “Immunological challenges in gene and cell therapy”
• Nathalie Cartier-Lacave, FR “Gene therapy for Huntington’s disease and spinocerebellar ataxias: from preclinical proofs of concept to first Phase 1 clinical trial”

Panel discussion: All speakers and patient representative (Oxana Illiach – CORD)

Panel Discussion

The Anatomy of an Acquisition: Bayer and AskBio
Available On Demand

The acquisition of AskBio is a historical deal, not only because of the size of the deal, but also its strategic relevance. With Bayer consolidating its presence in the Cell and Gene Therapy space, partnering with AskBio presents a unique opportunity to lead the genomic medicine space. There are multiple elements that made this deal a success: record-breaking deal-making speed during a global pandemic, joint development of milestones that align interests, and a unique operating model that safeguards AskBio’s independence are just a few of the critical success factors.

Join AskBio CEO and co-founder Sheila Mikhail and Marianne De Backer, EVP, Head of Strategy, Business Development & Licensing and Executive Committee Member at Bayer AG Pharmaceuticals, for a fireside chat and learn more about the recent acquisition.

To register for Biotech Showcase Digital, visit https://informaconnect.com/biotech-showcase/.

Join us at the virtual 2nd Annual Gene Therapy for Neurological Disorders conference from December 8-10, 2020 to learn about translational drug development challenges encountered in the development of neurological gene therapies.

December 10, 2020

10:00am ET

Lessons from the Clinic & Why AAV Biology Still Matters

Jude Samulski, PhD, Chief Scientific Officer, AskBio

Synopsis
• Follow the history of AAV administration for CNS disorders
• Discuss the value of AAV biology
• Review clinical data

11:20 am 

Q&A Panel Discussion: Delivery

Martin Marsala, Professor, University of California, San Diego
Krystof Bankiewicz, MD, PhD, Department of Neurological Surgery, The Ohio State University Wexner Medical Center
Olivier Danos, Chief Scientific Officer, REGENXBIO
R. Jude Samulski, Chief Scientific Officer, AskBio

Synopsis
• Comparing different patient populations for different indications and their suitability for different delivery routes
• Discussing bio-distribution vs. invasive procedures for delivery to the brain
• Exploring the risk/benefit of different routes of administration, is the data translatable?
• Introducing technology advances for the delivery devices – where is there room for improvement?

Join us at the 8th annual Chief Medical Officer Summit, the largest gathering of physicians in biotech, to be held virtually from October 26-27, 2020.

AskBio CMO, Martin (Casey) Childers, DO, PhD, will moderate a panel featuring CMOs from Epizyme, Annexin Pharmaceuticals and Aura Biosciences.

New Skills Required for CMOs

• A panel discussion of CMOs with different backgrounds about what skills they needed to learn for the role and how they learned them
• Support systems and networks for CMOs

October 27, 2020
2:45pm-3:25pm ET

AskBio is a proud sponsor of the 2020 Cell & Gene Meeting on the Mesa. CEO Sheila Mikhail will deliver a pre-recorded company presentation that will be available on demand to registered attendees from October 12-16, 2020.

AskBio will present on Tuesday, Oct 6, at the Chardan 4th Annual Genetic Medicines Conference, which will be held as a virtual event Oct 5-6, 2020.

Presentations

KOL Fireside Chat
Jude Samulski, PhD | President, Chief Scientific Officer and Co-founder
Oct. 6, 9:30am – 10:15am ET

Company Overview
Sheila Mikhail, JD, MBA | CEO and Co-founder
Oct. 6, 1:30pm – 1:55pm ET

The conference will have presentations from over 70 public and private companies who are leading the way in the genetic medicines space. The conference will feature presentations, fireside chats, panels, Q&A and 1×1’s. To learn more about the conference and the confirmed presenting companies, please contact CorpAccess@Chardan.com.

AskBio CEO Sheila Mikhail will participate on a panel discussion among biopharmaceutical leaders at the Longwood Healthcare Leaders Forum – Fall 2020 to be held on Monday, October 5, 2020.

Monday, October 5, 5:40 pm ET
IDENTIFYING NOVEL TECHNOLOGY

Sheila Mikhail, CEO, Asklepios BioPharmaceutical
Manuel Aivado, CEO, Aileron Therapeutics
Junichi Koga, Global Head, R&D, Daiichi Sankyo
John Reed, EVP and Global Head, R&D, Sanofi
Oliver Rosen, CMO, SQZ Biotech
Moderator: Uwe Schoenbeck, CSO, WWRD, Pfizer

AskBio CEO Sheila Mikhail will present on Friday, Oct 2, from 10:30-10:55am ET at the Jefferies Gene Therapy/Editing Summit 2020, which will be held as a virtual meeting.

AskBio CEO Sheila Mikhail will present at the Morgan Stanley Virtual 18th Annual Global Healthcare Conference taking place Monday, September 14 – Friday, September 18, 2020.

Fireside Chat

Friday, Sept. 18
9:30am ET

The Advanced Therapies Congress and Expo 2020, which is being held virtually, has been rescheduled to Sept. 8-11, 2020.

PRESENTATION
September 8
11:30am-12:00pm
Maximizing Value from the Expression Cassette in Gene Therapy Product Design
Speaker: David Venables, PhD, President, AskBio Europe

• Expression cassette design attributes
• Synthetic promoter design and validation
• Regulatable gene expression

We’re presenting at the 12th Annual Bioprocessing Summit, which will be held virtually from Aug 24-28, 2020.

Presentation

Track: Gene Therapy Manufacturing
Friday, Aug 28
12:20-12:40pm ET

Jacob Smith, PhD, Director, Process Development
Advances in rAAV Manufacturing: Vertical Integration and Continuous Process Improvement

Recombinant adeno-associated virus (rAAV) has emerged as a dominant gene-delivery vector of choice. Despite numerous advances in the clinic, the efficiency and cost of producing rAAV drug product to meet a rapidly growing industry has significant room for improvement. AskBio has developed a model for advancing multiple clinical programs, with an emphasis on establishing early benchmarks in manufacturing feasibility, while focusing on continuous process development and improvements to large-scale manufacturing.

AskBio CEO Sheila Mikhail will participate in a fireside chat presentation at the William Blair Biotech Focus Conference, which will be held as a virtual event August 4-6, 2020.

Presentation

Thursday, Aug. 6
2:00-2:45pm ET

Register today for The Art of Gene Therapy Digital Summit, a live two-day event on July 28-29, 2020, featuring industry-leading gene therapy companies and regulatory agencies such as the FDA, AskBio, Orchard Therapeutics, bluebird bio, REGENXBIO and many more.

The digital summit will shine a light on gene therapy future technologies, technical challenges, lessons learned from scale up to commercialization and what is on the horizon for regulatory guidelines.

Presentation

Wednesday, July 29
11:00am – 12:00pm ET
The Next Decade of Gene Therapy: Q&A panel discussion

AskBio’s Andy Holt, VP Business Development, joins a panel of gene therapy leaders from REGENXBIO, Orchard Therapeutics, In Vivo and MilliporeSigma to discuss what’s next in the industry.

We’re speaking at the Gene Therapy for Neurological Disorders Europe conference, which will be held as a virtual meeting on July 15, 2020. The conference unites industry leaders to help them overcome translational challenges and successfully bring CNS gene therapy candidates into the clinic.

PRESENTATIONS

9:40 am Case Study: Linking Biological Understanding to Determine Optimal Dose
Nathalie Cartier-Lacave, Founder & Chief Scientific Officer, BrainVectis (acquired by AskBio in April 2020)

• Linking dosage to what is known about the disease and the level of gene expression required
• Addressing translational challenges of extrapolating animal dosage predictions and demonstrating optimal dose
• Demonstrating a good mechanistic route between dose, mechanisms of action and endpoints. How precise can we be with our dosing?

11:10 am Assessing Different Vector Types for CNS Disorders & Promoter Choice
Michael Roberts, Chief Technology Officer, AskBio, Europe

• Reviewing different types of gene therapy and their suitability for neurological disorders
• Discussing the properties of vector design and novel gene control mechanisms
• Exploring appropriate promoter choice for CNS indications

12:45 pm PANEL DISCUSSION: Navigating the Regulatory Challenges for Neurological Gene Therapy Development
Nathalie Cartier-Lacave, Founder & Chief Scientific Officer, BrainVectis
Elena Hernandez Martinez de Lapiscina, Scientific Officer & Clinical Neurologist, Office of Therapies for Neurological & Psychiatric Disorders, European Medicines Agency
Hans-Juergen Fuelle, VP, Head Global Regulatory Strategy, AveXis
Victoria Palmi Reig, Product Lead, Advanced Therapy Medicinal Products, European Medicines Agency

• Exploring standardization of approaches in gene therapy studies
• Assessing geo-differences for regulatory challenges
• Reviewing new FDA guidelines and understanding Europe regulators point of view
• How do regulators view the development of gene therapies for ultra-orphan indications?

3:40 pm PANEL DISCUSSION: Rare vs Common Neurological Diseases
Krystof Bankiewicz, President & CEO, Brain Neurotherapy Bio, Inc.
Petra Kaufmann, SVP Translational Medicine & Clinical Development, AveXis
Claire Gelfman, VP, Pharmaceutical Development, Adverum Biotechnologies

• Discussing the current clinical programs and review of approved candidates – challenges and successes in rare neurological disorders
• Evaluating the opportunity for gene therapy for neurodegenerative disorders such as Parkinson’s Disease and Alzheimer’s Disease
• Considering route of administration and patient population size, is gene therapy a viable option for Alzheimer’s Disease and Parkinson’s Disease?

5:25 pm The Evolution of Gene Therapy for Neurological Disorders
Krystof Bankiewicz, President & CEO Brain Neurotherapy Bio, Inc. (Partnered with AskBio)

• Can gene therapy influence underlying neuropathological changes associated with CNS disorders?
• Reviewing the importance of gene delivery and how it has evolved over the decades
• Gene therapy, over the fence and into the commercialization forest – how to scale it up?

We’re proud to participate in the BIO International Conference, which has transitioned into a new, virtual event format for 2020, BIO Digital. This year, we were selected as one of two Buzz of BIO winners. The conference runs from June 8-12, 2020.

Don’t miss our on-demand sessions:

Company presentation
Andy Holt, VP Business Development & Manufacturing

PANEL: The Role of New Technologies in Enabling Cell & Gene Therapies
BioProcess International Theater
A panel of experts will discuss product development and manufacturing challenges for cell and gene therapies and technologies that are being developed to address these challenges. Panelists include end users that can discuss the needs for new technologies, as well as technology providers.

The panel will discuss technologies for:
• Scalable and cost efficient production of viral vectors for gene therapy
• Non-viral approaches for gene therapy
• Scaling up to meet demand for autologous cell therapies

Chair: Doug Miller, Managing Director, BPTG
Panelists:
Daniella Kranjac, Co-Founder and Managing Director, Dynamk Capital
Tim Kelly, President, Manufacturing, AskBio
Andy Ramelmeier, Executive Vice President, Technical Operations, Sangamo Therapeutics
Ran Zheng, Chief Technical Officer, Orchard Therapeutics

AskBio CEO Sheila Mikhail will present at the Jefferies Virtual Healthcare Conference being held June 2-4, 2020. This virtual gathering of over 400 public & private healthcare companies and 2,500 leading executives, institutional investors, private equity investors & VCs will address near- and long-term investment opportunities and discuss the current mechanisms driving healthcare in the U.S. and internationally.

AskBio Presentation
June 2
9:35-9:55am ET

Join us on Tuesday, May 26, 2020, for Chardan’s 4th Annual Genetic Medicines Manufacturing Summit, which will be held in a virtual format this year. This event runs from 8:30am-5:00pm ET. Register at the link above, or email corpaccess@chardan.com with questions and 1×1 meeting requests.

Fireside Chat
12:45-1:45pm ET
Tim Kelly | President, Manufacturing – AskBio
Andy Holt | VP, Business Development & Manufacturing, AskBio
Gbola Amusa | Chardan Partner, Head of Healthcare Research

SUMMARY OF THE SUMMIT: 
Hear from key leaders involved in genetic medicines manufacturing, including AskBio, CureVac, ElevateBio, Homology Medicines (FIXX), MeiraGTx (MGTX), Moderna (MRNA), Prevail Therapeutics (PRVL), Translate Bio (TBIO), uniQure (QURE), and univercells. These companies will address various topics including:

1. Learn what scaling up manufacturing entails across different platforms including mRNA, AAV vector, and cell-based therapies.
2. Learn how developers of vaccines and therapies for Covid-19 are scaling up manufacturing to meet global demands (e.g. including in Emergency Use Authorization scenarios), and understand the read-across for other pipeline assets.
3. Understand how companies are addressing new challenges posed by the Covid-19 pandemic with respect to supply chain management, securing clinical material and other logistics.
4. Discuss different technologies for AAV manufacturing and how the technologies can be deployed to meet the needs for different indications in terms of scale, efficiency and purity.
5. Understand the advantages and challenges of developing internal manufacturing vs. working with an external partner.

We’re looking forward to the ASGCT 2020 Annual Meeting, which will be a virtual conference held May 12-15, 2020. We have a number of oral and poster presentations scheduled.

ORAL PRESENTATIONS

Adeno-Associated Virus Evolution from “Almost a Virus” to “An Amazing Vector”
Presenter:  Jude Samulski, PhD, UNC Gene Therapy Vector Core, AskBio
Tuesday, May 12, 2020
Main Session:  The Golden Age of Gene Therapy:  Past, Present & Future Perspectives
Presentation Time:  8:00am – 8:26am ET

Phase 1 Study of Gene Therapy in Late-Onset Pompe Disease: Analyses of Safety and Secondary Endpoints
Presenter:  Dwight Koeberl, MD, PhD, Duke University School of Medicine
Wednesday, May 13, 2020
Main Session:  AAV Vectors – Clinical Studies II
Presentation Time:  5:15pm – 5:30pm ET
Abstract number:  509

AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo
Presenter:  Sara Powell, PhD, AskBio
Wednesday, May 13, 2020
Session Title:  Controlling AAV Gene Expression: Shifting Paradigms
Presentation Time: 4:00pm – 4:15pm ET
Abstract number: 511

POSTER PRESENTATIONS

Tuesday, May 12, 2020

Full or Empty: A CMC Perspective on Production and Purification of Empty Vector Particles
Presenter:  Paul Greback-Clarke, AskBio
Session Title:  Vector and Cell Engineering, Production or Manufacturing
5:30pm – 6:30pm ET
Abstract number: 465

Wednesday, May 13, 2020

Development and Validation of a CE-SDS Method for Purity Determination of AAV8
Presenter:  Jeremy Johnston, MS, AskBio
Session Title:  AAV Vectors – Virology and Vectorology
5:30pm – 6:30pm ET
Abstract number: 554

Development of a Molecular Switch to Regulate Cas9 Expression
Presenter:  Bikash Shakya, PhD, AskBio
Session Title:  Gene Targeting and Gene Correction
5:30pm – 6:30pm ET
Abstract number: 644

Thursday, May 14, 2020

Novel Machine Learning Algorithms Improve the Design of Synthetic Promoters Resulting in More Selective and Higher Levels of Gene Expression in Muscle and Liver Cells
Presenter:  Michael Roberts, PhD, AskBio
Session Title:  AAV Vectors – Virology and Vectorology
5:30pm – 6:30pm ET
Abstract number:  1003

Development of Novel Synthetic Promoters for CNS Gene Therapy
Presenter:  Riccardo Privolizzi, PhD candidate, University College of London
Session Title:  Vector and Cell Engineering, Production or Manufacturing
5:30pm – 6:30pm ET
Abstract number:  1262

The Alliance for Regenerative Medicine (ARM) leadership has canceled the 2020 Cell and Gene Meeting on the Mediterranean. This event will not take place until April 7-9, 2021 in Barcelona.

OCT Southeast has been postponed. Stay tuned for new dates!

Learn how to build your clinical trials around patients with AskBio’s Joy Carson, VP Clinical Development & Operations, at Outsourcing in Clinical Trials (OCT) Southeast 2020. She will present at two sessions.

Date & time pending conference rescheduling.
Building your study around the patient to promote overall success within trials

• Utilizing various methods of patient engagement such as apps, websites and text messages to ensure the patient is well informed throughout trial
• Easing the financial burden of trial on patients due to traveling by utilizing decentralized methods to encourage participation from early stages to completion
• Understanding the importance of ensuring diversity within trials to ensure inclusion of patients from various backgrounds
• The importance of technology in fostering a more patient minded approach to clinical trials by allowing decentralization of the clinical trial process

Date & time pending conference rescheduling.
Speaker Hosted Roundtable – RT1
The role of patient registries to assess safety risks in clinical trials

This interactive roundtable session offers a unique opportunity to come together with your peers to share best practices and develop solutions to critical challenges facing the industry as a whole. Hosted by Joy Carson, this session is an interactive way to build your personal network and learn from the experience and expertise of others.

This meeting has been postponed. A new date has not yet been scheduled.

We’re a proud sponsor and presenter at the Gene Therapy in Edinburgh symposium, which is being held at Queen’s Medical Research Institute on March 26, 2020 from 10:00am to 5:45pm. The symposium is an opportunity to bring together researchers across Edinburgh who are interested in gene-based therapies. There is no charge to attend, but registration is required here.

PRESENTATION
Engineering Tightly Controlled and Tuneable Gene Cassettes for Therapeutic Applications
Speaker: Michael Roberts, PhD, Chief Technology Officer, AskBio Europe
3:45pm-4:15pm

The 2020 MDA Clinical & Scientific Conference in Orlando, FL has been postponed. At this time, a new date is still pending.

PANEL: Leading Emerging Growth Companies: A CEO Perspective

This conference has been postponed, and planning for a later date in 2020 is underway.

AskBio CEO Sheila Mikhail will participate in a panel discussion CED Venture Connect 2020. This CEO panel will explore the leadership ups and downs associated with being at the helm of an emerging-growth life science company.

Speakers
– Sheila Mikhail, CEO & co-founder, AskBio
– Sapan Shah, CEO, Stride Bio
– Michael Solomon, CEO, Ribometrix

David Favre, VP Translational Medicine, will present at the EIP Symposium in Lisbon, which will be held February 17-19, 2020.

AAV Immunogenicity: Novel Vectors & New Questions
Tuesday, Feb. 18
10:00am-10:45am

Cell and gene therapies are rapidly developing as novel therapeutic options for patients with unmet medical needs. These encompass rare genetic diseases, including primary immune deficiencies (e.g. ADA-SCID), metabolic diseases (e.g. retinopathies, hemophilia, lysosomal storage diseases), muscular dystrophies (e.g. DMD, SMA), hemoglobinopathy (e.g. b-thalassemia) and neurodegenerative disorders (e.g. Alzheimer’s, Parkinson’s, Huntington’s diseases). It now extends to non-monogenic disease areas, such as chronic inflammatory diseases (e.g. arthritis), cardio-vascular diseases (e.g. congestive heart failure) and refractory/relapsed cancers (e.g. TCR/CAR-T cell therapies).

AAVs hold a unique position as one of the most effective and safe vectors for direct in vivo gene delivery of therapeutic genes. The biology, pharmacology and immunogenicity of AAV gene delivery in vivo are essential for success. As AAV vectors progress along with new strategies to dose or re-dose subjects with pre-existing immunity, new questions will arise on the safety and immunogenicity of novel vectors. AskBio will discuss the opportunities and challenges of novel AAVs from an immunogenicity standpoint.

Sheila Mikhail – CEO, President, Co-Founder

AskBio CEO Sheila Mikhail was recognized at Springboard Enterprises’ 13th Annual Winners’ Circle Dinner in New York City on October 23rd, as the 2019 Northstar honoree. The award is given annually to high-performing woman business entrepreneurs who are transforming their industries. Springboard’s mission is to accelerate the growth of entrepreneurial companies led by women through access to essential resources and a global community of experts.

Sheila Mikhail – CEO, President, Co-Founder

AskBio CEO Sheila Mikhail presented at the 3rd annual Chardan Genetic Medicines Conference in New York on October 7, 2019. As one of 60 private and publicly traded companies invited to participate, AskBio was the only one with an end-to-end gene therapy platform. Sheila’s presentation was viewed by leading bioscience and major pharma companies as well as researchers and academics from around the globe.

Sheila Mikhail – CEO, President, Co-Founder

The Cell & Gene Meeting on the Mesa is the sector’s foremost annual conference, bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Sheila Mikhail, CEO, of AskBio presented our differentiating capabilities, technology and therapeutic pipeline. Also attending was David Venables, who served as Synpromics president and now will lead our AskBio European operations.

Joy Carson – AskBio’s Vice President of Clinical Development & Operations
Sharon Hesterlee, PhD – EVP Portfolio Development

Joy Carson, AskBio’s Vice President of Clinical Development & Operations, and Sharon Hesterlee, AskBio’s Executive Vice President Portfolio Development, joined patients, families and leading stakeholders at the first-annual National Limb Girdle Muscular Dystrophy Conference in support of the LGMD patient community in Chicago. The event was attended by the nation’s leading neurologists in Limb Girdle Muscular Dystrophy along with leading biotech companies on the cutting edge of emerging research.

Jude Samulski – Co-Founder
Sharon Hesterlee, PhD – EVP Portfolio Development

AskBio’s Jude Samulski and Sharon Hesterlee joined the 25th Parent Project Muscular Dystrophy in Orlando. Families, physicians, researchers, caregivers, industry partners, and those living with Duchenne gathered to discuss the latest research and policies surrounding the disease. AskBio is proud to be part of the conversation and is working hard to support gene therapy treatment.

Andy Holt, AskBio’s VP of Business Development, joined the panel discussion on gene therapy hosted by the Alliance for Regenerative Medicine in April 2019.

Josh Grieger, PhD – Chief Technology Officer