Dae Gon Ha

Jack Allen

Philip Nadeau

Yanan Zhu

Huidong Wang

Luca Issi

Lut Ming Cheng

Brian Cheng

Cheng Li

Eric Schmidt

Gena Wang

Greg Harrison

Jay Olson

Jingming Chen

Joon Lee

Julien [on for Liisa Bayko]

Mary Kate Davis

Mehdi Goudarzi

Mia Bongiorno

Nicholas Econom

Samantha Semenkow

Steven Seedhouse

- The conference emphasized the **coexistence and evolution of diverse gene editing technologies**, with a focus on product profile and directing tools to their most effective applications.  **[0001650664_2193544_2]** 
- **Delivery mechanisms are critical**, with LNPs being a leading in vivo method, though requiring optimization. Non-viral approaches are highlighted for their potential to achieve **lower cost of goods** and enhance commercial viability.  **[0001650664_2193544_3]** **[0001650664_2193544_4]** **[0001650664_2193544_9]** 
- Companies reported **strong clinical progress and commercial viability** for one-time gene editing therapies, exemplified by Editas Medicine's **EDIT-401** achieving a **90% reduction in LDL C** and targeting human Proof-of-Concept by **2026**.  **[0001650664_2193544_0]** **[0001650664_2193544_8]** 
- Several participants provided pipeline updates, including Yultech Therapeutics with **4-5 programs in the clinic** and a Phase 3 program starting early next year (2026)  **[0001650664_2193544_0]** , and Gen Edit Bio having dosed **4 patients** in an IIT for TGFBI mutant corneal dystrophy with an FDA IND filing planned by year-end (2025).  **[0001650664_2193544_1]**

Chardan's 9th Annual Genetic Medicines Conference Highlights Advancements in Gene Editing Therapies

- Editas is primarily focused on its **EDIT-401 LDLR program**, an in vivo gene editing therapy designed to achieve a **90% reduction in LDL-C**.  **[0001650664_2181296_0]** 
- The company plans to file an Investigational New Drug (IND) or Clinical Trial Application (CTA) for EDIT-401 by **mid-2026** and anticipates having **human proof of concept data by the end of 2026**.  **[0001650664_2181296_0]** **[0001650664_2181296_5]** 
- Editas emphasizes the **differentiated efficacy** of its upregulation strategy, which has shown a six-fold increase in LDL receptor levels in nonhuman primates, leading to a 90% LDL-C reduction, surpassing the 40-60% reduction seen with current standard-of-care treatments.  **[0001650664_2181296_1]** **[0001650664_2181296_2]** 
- The company has a cash runway that extends into **Q2 2027**, which is expected to fund the IND/CTA filing, human proof of concept, and further development.  **[0001650664_2181296_5]** 
- The initial target market for EDIT-401 is expected to be **refractory heterozygous familial hypercholesterolemia patients**, with potential for expansion into broader hypercholesterolemia populations as long-term safety is established.  **[0001650664_2181296_3]**

Editas Provides Update on EDIT-401 LDLR Program and Strategic Focus

- Editas Medicine, Inc. reported **in vivo preclinical proof-of-concept data** for **EDIT-401**, an experimental gene-editing therapy aimed at significantly reducing LDL-cholesterol (LDL-C).  **[1650664_0001650664-25-000148_esgct_datareleasexfinal.htm:0]** **[1650664_0001650664-25-000148_edit-20251009.htm:2]** 
- Preclinical studies demonstrated **≥90% mean LDL-C reduction** in non-human primates within 48 hours of a single dose and in mouse models.  **[1650664_0001650664-25-000148_esgct_datareleasexfinal.htm:0]** **[1650664_0001650664-25-000148_edit-20251009.htm:2]** 
- The LDL-C reduction was **maintained in mouse models** in a three-month study, indicating a durable effect.  **[1650664_0001650664-25-000148_esgct_datareleasexfinal.htm:0]** **[1650664_0001650664-25-000148_edit-20251009.htm:2]** 
- The therapeutic strategy involves a **CRISPR/Cas9 nuclease and dual gRNAs with LNP delivery** to upregulate the LDL receptor (LDLR), resulting in a **≥6-fold mean increase in LDLR protein** in the non-human primate liver.  **[1650664_0001650664-25-000148_esgct_datareleasexfinal.htm:0]** **[1650664_0001650664-25-000148_edit-20251009.htm:2]**

Editas Medicine Reports Positive Preclinical Data for EDIT-401

- Editas Medicine reported **_in vivo_ preclinical proof-of-concept data** for its experimental therapy, **EDIT-401**, at the 32nd Annual European Society of Gene and Cell Therapy (ESGCT) Congress on **October 9, 2025**.  **[1650664_84ac76fad9864748807562a46be978a6_0]** 
- EDIT-401 is a potential one-time therapy designed to **significantly reduce LDL-cholesterol (LDL-C)** through the upregulation of the LDL receptor (LDLR).  **[1650664_84ac76fad9864748807562a46be978a6_0]** 
- Preclinical studies demonstrated **robust efficacy**, achieving **≥90% LDL-C reduction** in non-human primates within 48 hours of a single dose, and a **durable effect** maintained in mouse models over a three-month study.  **[1650664_84ac76fad9864748807562a46be978a6_0]**

Editas Medicine Reports Preclinical Proof-of-Concept Data for EDIT-401

- **Editas Medicine** nominated **EDIT-401** as its lead in vivo development candidate, an experimental gene editing medicine targeting hyperlipidemia.  **[1650664_20250925LN83231:2]** 
- **EDIT-401** achieved approximately **90% mean LDL cholesterol reduction** with a single dose in non-human primate studies, significantly outperforming standard-of-care therapies.  **[1650664_20250925LN83231:2]** 
- The company expects to achieve human proof-of-concept data by the **end of 2026** and plans to submit an investigational new drug application by **mid-2026**.  **[1650664_20250925LN83231:2]** 
- As of **June 30, 2025**, Editas Medicine reported **$178.5 million in cash, cash equivalents, and marketable securities**, providing an operational runway into the **second quarter of 2027**.  **[1650664_20250925LN83231:3]**

Editas Medicine Nominates Lead Gene Editing Candidate EDIT-401 and Updates Financial Position

Editas Medicine (EDIT)