- **Precision Biosciences** and **eCure** are advancing gene insertion therapies, with eCure reporting positive **10-month clinical data** for its **ornithine transcarbamylase (OTC) deficiency** program using Precision's **Arcus** technology, demonstrating **significant clinical improvements** in a pediatric patient.  **[0001357874_2194268_0]** **[0001357874_2194268_4]** 

| Metric | At Dosing | 10 Months Post-Treatment |
|---|---|---|
| Patient Age | **6.5 months**  **[0001357874_2194268_4]**  | N/A |
| Scavenger Medications | On standard of care  **[0001357874_2194268_4]**  | Discontinued within 3 months  **[0001357874_2194268_4]**  |
| Hyperammonemic Events/Crisis | Experienced  **[0001357874_2194268_4]**  | None  **[0001357874_2194268_4]**  |
| Ammonium Levels Reduction | N/A | **~50%**  **[0001357874_2194268_4]**  |
| Liver Transplant List | On list  **[0001357874_2194268_4]**  | Off list  **[0001357874_2194268_4]**  |

- Precision Biosciences is also developing **Arcus nuclease-based gene editing** for **hepatitis B**, delivered via **lipid nanoparticles (LNP)**, with initial clinical data showing **good safety** and **antiviral activity** (substantial S antigen reductions) in the lowest dose cohort.  **[0001357874_2194268_7]** 
- Companies like **Seamless Therapeutics** and **Harborside** are leveraging **recombinase technology** for gene integration, offering advantages for inserting large DNA fragments and targeting specific genomic safe harbors or endogenous promoters, which is crucial for treating diseases with multiple mutations and ensuring durable expression in growing tissues.  **[0001357874_2194268_1]** **[0001357874_2194268_2]** 
- The overall sentiment in the genetic medicine space is cautiously optimistic, with a strong desire for **more clinical data** and a focus on **commercial potential** and addressing unmet needs, with regulators showing flexibility and support for advanced therapies.  **[0001357874_2194268_10]**

Chardan Conference Highlights Advances in Gene Editing and Clinical Progress

- **Precision Biosciences** presented its proprietary **Arcus gene editing platform**, highlighting its differentiation from other technologies due to its **compact size** and **single-protein simplicity**.  **[0001357874_2194265_0]** **[0001357874_2194265_1]** 
- The company's lead program, **PBG-HBV**, is designed to achieve a **complete cure for Hepatitis B** by directly eliminating cccDNA and inactivating integrated HBV DNA, a distinct approach from current functional cure strategies.  **[0001357874_2194265_2]** **[0001357874_2194265_3]** **[0001357874_2194265_9]** 
- In the ongoing **ILLUMINATE B Phase 1 study**, **Cohort 1 (0.2 mg/kg)** demonstrated **good tolerability** and **antiviral activity**, with all three participants achieving **45% to almost 70% S antigen reduction**.  **[0001357874_2194265_5]**  One participant sustained a **50% reduction in S antigen seven months post-treatment**.  **[0001357874_2194265_5]** 
- **Cohort 2 (0.4 mg/kg)** has also shown **good tolerability** with repeat administrations, and **Cohort 3 has been initiated**.  **[0001357874_2194265_5]** 
- Precision Biosciences plans to share **additional data** from the ILLUMINATE B study at the upcoming **AASLD Liver Meeting** and will continue to optimize dosing schedules, potentially exploring shorter dosing intervals and higher dose levels.  **[0001357874_2194265_9]**

PRECISION BIOSCIENCES (DTIL)