7:00 am Check-In & Coffee

7:50 am Chair’s Opening Remarks

Accelerating Innovative Payload Design

8:00 am An Overview of the Current Landscape of Next-Generation Gene Therapy Vectors

Synopsis

  • Summarizing the current split of different vector types across the gene therapy landscape
  • A deep dive into preclinical trends, looking at which vector types are increasing in prevalence
  • Outlining the current deal & acquisition landscape of the field

8:30 am Cassette Engineering to Improve Safety & Expression Profiles

  • Darin Falk Chief Scientific Officer, Lacerta Therapeutics, Inc.

Synopsis

  • Promoter optimization to accommodate genome size and expression profile
  • Incorporation of regulatory elements to avoid over-expression related toxicity
  • Strategies to predict activity and promote translational outcomes following cassette optimization

9:00 am Driving Towards On-Off Transgene Expression

  • Daniel Cohen Senior Director, Head of Vector Optimization, Spark Therapeutics

Synopsis

  • Evaluating tissue and cell specificity of promoters
  • Exploring programmable promoters
  • Leveraging approaches to achieve controllable transgene expression

9:30 am Morning Break & Networking

10:15 am Optimization of AAV Vector Genome Design for the Treatment of Motor Neuron Diseases

  • Jun Xie Director - vector development of viral vector core, UMass Medical School

Synopsis

  • Overview of the AAV gene therapy on SMA and ALS
  • Optimized vector genome Improves the efficacy and safety of AAV9-mediated gene therapy for SMA
  • Optimized artificial miRNAs by AAV9 vectors drastically improve survival and function in ALS mice

Beyond AAV: Highlighting Boundary-Pushing Vector Platforms

10:45 am Prometheus: The First Adjustable Dose Gene Therapy Platform Technology

Synopsis

  • An engineered gene therapy by design: ultra-low-cost, non-antigenic, specific, localized, and exceptionally well tolerated
  • Overcoming the challenges of gene therapy’s fixed dose nature via a novel dose adjustment mechanism
  • Enabling the replacement of subcutaneous protein treatments, with zero-order kinetics delivery

11:15 am Epigenetic Editing with CRISPR-GNDM: MDL-101 is a Novel Muscle-Tropic AAV Vector for the Treatment of LAMA2-CMD

  • Tetsuya Yamagata Scientific Advisory & Chief Scientific Officer, Modalis Therapeutics

Synopsis

  • MDL-101 demonstrates promising efficacy in dyW mouse disease model of LAMA2-CMD, with optimal vector biodistribution, robust LAMA1 expression and significant improvement in the disease phenotype
  • MDL-101 demonstrates a range of safety profiles in NHPs, exhibiting strong biodistribution to skeletal muscles, robust expression of GNDM, and therapeutically significant induction of LAMA1 mRNA and protein
  • With the successful completion of Process Development for MDL-101 vector production and IND-enabling discussions with the FDA, MDL-101 stands poised to advance toward clinical development

11:45 am Harnessing Innovation in Lentiviral Vector Platforms

  • Alessio Cantore Group Leader, San Raffaele-Telethon Institute for Gene Therapy

Synopsis

  • Presenting a next-generation lentiviral platform vector system: what distinguishes it from the first generation?
  • How have engineering efforts innate immune responses and gene transfer efficiency?
  • Establishing future research needs to further increase safety and efficacy

12:15 pm Lunch & Networking

1:15 pm Engineering ARRDC1-Mediated Microvesicles (ARMMs) for Targeted Non-Viral Delivery of Large Payloads

Synopsis

  • ARMMs are modular vehicles for delivery of Cas/gRNA RNPs, RNA, and effector proteins
  • ARMMs have a differentiated biodistribution to a range of tissue and cell types
  • ARMMs can be surface engineered with targeting moieties to direct tropism to specific cell types

1:45 pm Advancing a New Class of Precision Genetic Medicine with 3DNA Nanoparticles

  • Alex Tracy Chief Technical Officer, Code Biotherapeutics

Synopsis

  • Introduction to the 3DNA platform – critical design elements that contribute to its functionality
  • Building a scalable, modular and robust manufacturing process to enable more access to genetic medicines
  • Future directions for DNA nanoparticles (DNPs) within the genetic medicine landscape with a focus on cell-specific targeting and redosability

2:15 pm Non-Replicative HSV Vectors for Prevalent Chronic Diseases in Neurology

Synopsis

  • Pinpoint delivery to the nervous system: PNS and CNS
  • Addressing prevalent disease in neuro-urology: preclinical safety and efficacy data
  • Scalable and efficient manufacturing

2:45 pm Afternoon Refreshment & Networking Break

Vector Design to Enable Scalable, Cost-Effective Manufacturing

3:15 pm Beyond AAV: Unlocking the Potential of Gene Therapy with Novel Parvovirus Vectors

  • Marc Abrams Chief Scientific Officer, Carbon Biosciences

Synopsis

  • New recombinant non-AAV parvovirus vectors designed to overcome many of the challenges associated with AAV vectors
  • CBN-1100 is a vector derived from a natural parvovirus species featuring higher cargo capacity compared to AAV, potency in diverse cell types, a compelling liver de-targeted distribution profile, high manufacturability and scalability, and minimal human seroprevalence
  • The pharmacological attributes of CBN-1100, including in nonhuman primates, demonstrate that viral vectors powered by natural non-AAV parvovirus capsids offer a path forward for numerous targets and indications that are undruggable using current gene therapy technology

3:45 pm Panel Discussion: Developing Robust, Clinical Stage-Ready CMC Processes

  • Alex Tracy Chief Technical Officer, Code Biotherapeutics
  • Jing Liao Director - Vector Operations, Alexion Pharmaceuticals

Synopsis

  • Ensuring vector design and selection is optimized for future manufacturing needs
  • Discussing cost-effectiveness vector development strategies to improve yields and quality of novel vector platforms
  • Weighing up whether to develop in-house manufacturing capabilities or outsource
  • Outlining what good teamwork with process development and CMC teams looks like

4:15 pm Chair’s Closing Remarks

4:20 pm End of Conference