Generation Bio Announces the Acceptance of Five Abstracts and an Invited Oral Presentation at the American Society of Gene and Cell Therapy 27th Annual Meeting

Generation Bio Co. (Nasdaq:GBIO) a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today announced that six abstracts highlighting preclinical data from its cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms have been accepted for presentation, including one oral presentation, at the American Society of Gene and Cell Therapy (ASGCT) 27 th Annual Meeting taking place May 7-11, 2024 in Baltimore, MD.

"We are delighted to have a robust presence at this year's ASGCT meeting and showcase some of the innovative technical work underpinning the breakthroughs we have achieved for our cell-targeted lipid nanoparticle (ctLNP) and immune-quiet DNA (iqDNA) platforms," said Matt Stanton, Ph.D., chief scientific officer. "We've made rapid progress across our platforms and look forward to presenting further updates at the meeting."

Oral Presentation

Title: Highly Specific, In Vivo Delivery to T-Cells with Cell-Targeted Lipid Nanoparticles
Session: Targeting Specific Cell Types with Nonviral Delivery
Session Location: Room 314-317
Session Date/Time: Saturday, May 11, 8:00 a.m. – 9:45 a.m. ET
Presentation Time: 8:35 a.m. – 9:10 a.m. ET

Poster Presentations

Title: iqDNA Is an Engineered DNA Cargo That Avoids Innate Immune Activation While Retaining Durable Transgene Expression
Session: Challenges to Immunological Responses to Therapeutic Interventions
Session Date/Time: Thursday, May 9, 2024, 12:00 p.m. ET – 7:00 p.m. ET
Abstract Number : 1294

Title: Rapid Enzymatic Synthesis and Scaling of DNA Gene Therapy Molecules
Session: Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates
Session Date/Time: Thursday, May 9, 12:00 p.m. – 7:00 p.m. ET
Abstract Number : 1232

Title: Optimization of Ionizable Lipid Structure to Modulate ApoE Binding and Enable Stealth Lipid Nanoparticles
Session: Other Nonviral Delivery
Session Date/Time: Thursday, May 9, 12:00 p.m. ET – 7:00 p.m. ET
Abstract Number : 1240

Title: Development of Cell-Targeted Lipid Nanoparticles Using Antibody Derived Ligands for Targeted Delivery to Hepatocytes and Immune Cells
Session: Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates
Session: Other Nonviral Delivery
Session Date/Time: Thursday, May 9, 12:00 p.m. ET – 7:00 p.m. ET
Abstract Number : 1241

Title: In Vivo High-Throughput Pooled Screening of Transcriptional Regulatory Elements for Gene Therapy
Session: Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates
Session Date/Time: Thursday, May 9, 12:00 p.m. ET – 7:00 p.m. ET
Abstract Number : 1233

The ASGCT abstracts are available to the public here . A copy of the presentation materials will be added to the "Our Scientific Presentations" section of the company's website here on the day of each presentation.

About Generation Bio

Generation Bio is innovating non-viral genetic medicines to provide durable and redosable treatments for hundreds of millions of patients living with rare and prevalent diseases. The company is developing two distinct and complementary platforms: a potent, highly selective cell-targeted lipid nanoparticle (ctLNP) delivery system and a novel immune-quiet DNA (iqDNA) cargo produced by a scalable capsid-free manufacturing process that uses proprietary cell-free rapid enzymatic synthesis (RES). With these platforms, Generation Bio aims to develop the next wave of non-viral genetic medicines to support its mission to extend the reach of genetic medicine to more people living with more diseases, around the world.

For more information, please visit www.generationbio.com .

Investors and Media Contact
Maren Killackey
Generation Bio
mkillackey@generationbio.com
857-371-4638


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- Oral presentation at ASGCT described selective, high levels of therapeutic transgene   delivery to T cells in vivo by cell-targeted lipid nanoparticle (ctLNP)

- ASGCT poster presentations described immune-quiet DNA (iqDNA) as partially single-stranded, produced by flexible, scalable, proprietary rapid enzymatic synthesis

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Generation Bio Announces the Presentation of Preclinical Data on iqDNA and ctLNP Platforms at the ASGCT 27th Annual Meeting

  • Data presented show immune-quiet DNA (iqDNA) is a partially single-stranded DNA cargo that maintains expression while evading innate immune sensors
  • Rapid enzymatic synthesis continues to improve iqDNA performance through process development and the identification of novel structural elements
  • Data on cell-targeted LNP (ctLNP) in vivo delivery of therapeutic transgenes to T cells to be discussed in an oral presentation on Saturday, May 11

Generation Bio Co. (Nasdaq:GBIO) a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, today presented five posters on its immune-quiet DNA (iqDNA) and cell-targeted lipid nanoparticle (ctLNP) platforms as well as its enzymatic synthesis manufacturing at the American Society of Gene and Cell Therapy (ASGCT) 27 th Annual Meeting. A sixth presentation on the company's ctLNP platform will be discussed in an oral presentation on Saturday, May 11.

"The data we're presenting at ASGCT showcase some important mechanistic underpinnings of our novel platforms, which we believe have the potential to overcome the core challenges facing the field of genetic medicine," said Matt Stanton, Ph.D., chief scientific officer of Generation Bio. "We believe immune-quiet DNA cargo and highly selective, targeted LNP delivery are the tools required to unlock the full potential of non-viral genetic medicines, and we are applying them to our current portfolio of programs in T cells, hematopoietic stem cells, and hepatocytes."

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