- **Electra Therapeutics** announced that its lead drug candidate, **ELA026**, has received **U.S. FDA Breakthrough Therapy designation (BTD)** and **European Medicines Agency (EMA) Priority Medicines (PRIME) designation** for the treatment of **secondary hemophagocytic lymphohistiocytosis (sHLH)**.  **[822370_cb0d86359eeb4e9d9e4fe4484eabf887_0]** 
- ELA026 is the **first investigational therapy** to receive both BTD and PRIME designations for sHLH, a **life-threatening hyperinflammatory disease** with significant unmet medical need.  **[822370_cb0d86359eeb4e9d9e4fe4484eabf887_0]** **[822370_cb0d86359eeb4e9d9e4fe4484eabf887_1]** 
- These designations are supported by **positive results from a completed Phase 1b study**, where ELA026 achieved **100% overall survival at 8 weeks**, **100% overall response rate by week 4**, and **100% hospital discharge** in patients with malignancy-associated HLH (mHLH).  **[822370_cb0d86359eeb4e9d9e4fe4484eabf887_0]** 
- Electra has initiated the **SURPASS global pivotal Phase 2/3 trial** for ELA026 in sHLH, which is currently enrolling and dosing patients.  **[822370_cb0d86359eeb4e9d9e4fe4484eabf887_0]**

Electra Therapeutics' ELA026 Receives Key Regulatory Designations

- Cellectis hosted an R&D Day on **October 16, 2025**, presenting promising Phase 1 clinical data for **lasme-cel (UCART22)** in relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL), demonstrating an **Overall Response Rate (ORR) of 68%** with Process 2 and **83%** at the Recommended Phase 2 Dose (RP2D).  **[822370_47c44c3354044b17bb7ea7b76d0fea20_0]** 
- The **BALLI-01 pivotal Phase 2** study for lasme-cel in r/r B-ALL has been initiated, with a Biologics License Application (BLA) anticipated in **2028**.  **[822370_47c44c3354044b17bb7ea7b76d0fea20_0]** **[822370_47c44c3354044b17bb7ea7b76d0fea20_3]** 
- Cellectis estimates **potential peak gross sales of up to ~$700 million** for lasme-cel across the U.S., EU4, and UK by 2035, with the possibility to reach **~$1.3 billion** with label expansion.  **[822370_47c44c3354044b17bb7ea7b76d0fea20_0]** **[822370_47c44c3354044b17bb7ea7b76d0fea20_4]** 
- Preliminary data for **eti-cel (UCART20x22)** in relapsed or refractory non-Hodgkin lymphoma (r/r NHL) showed an **Overall Response Rate (ORR) of 86%** and a **Complete Response (CR) rate of 57%**.  **[822370_47c44c3354044b17bb7ea7b76d0fea20_4]**

Cellectis Highlights Promising Lasme-cel Clinical Data and Commercial Opportunity

- **AB Science** announced initial positive data from the **Phase 1 study (AB18001, NCT05211570)** of its molecule **AB8939 in combination with venetoclax** for refractory or relapsed Acute Myeloid Leukemia (AML).  **[822370_062163a7d4bd44d383e35b0e79d6039d_0]** **[822370_062163a7d4bd44d383e35b0e79d6039d_1]** 
- The first three patients, all with very unfavorable genetic profiles (including TP53 mutation and complex karyotypes), showed a **100% disease control rate** and a **100% partial response rate** (including one complete remission) after the first 14-day treatment cycle.  **[822370_062163a7d4bd44d383e35b0e79d6039d_0]** 
- The combination treatment was **well-tolerated with no hematological toxicity**, and these patients were receiving third- or fourth-line treatment, with two having previously progressed on venetoclax.  **[822370_062163a7d4bd44d383e35b0e79d6039d_0]** 
- AB Science will hold a virtual conference on **Thursday, October 16, 2025, from 2pm to 3pm CET** to provide more details on these initial clinical results.  **[822370_062163a7d4bd44d383e35b0e79d6039d_0]** **[822370_062163a7d4bd44d383e35b0e79d6039d_1]** 
- AB8939 has been granted **Orphan Drug Designation** by both the European Medicines Agency (EMA) in April 2025 and the US Food and Drug Administration (FDA) for AML.  **[822370_062163a7d4bd44d383e35b0e79d6039d_3]**

AB Science Reports Initial Positive Phase 1 Data for AB8939 in AML

- **Neurogene** has completed discussions with the FDA on the protocol for its **Embolden registrational trial** of **NGN-401 gene therapy** targeting Rett syndrome.  **[822370_f44427099b5440c2aac2749ead0a1934]** 
- The company plans to **initiate dosing in the fourth quarter of 2025** across **13 sites** for the Embolden trial.  **[822370_f44427099b5440c2aac2749ead0a1934]** 
- Preclinical data demonstrated that **intracerebroventricular (ICV) delivery of NGN-401** achieves superior adeno-associated virus biodistribution in brain regions critical to Rett syndrome compared to intrathecal lumbar (IT-L) delivery.  **[822370_f44427099b5440c2aac2749ead0a1934]** 
- Neurogene is participating in the **FDA's START pilot program** and has received **RMAT designation**, positioning it for early discussions aimed at an expedited Biologics License Application (BLA) submission.  **[822370_f44427099b5440c2aac2749ead0a1934]** 
- Following the announcement of FDA protocol discussions and positive preclinical data, Neurogene's shares **rose 11% to $28.70**.  **[822370_f44427099b5440c2aac2749ead0a1934]**

Neurogene Plans Embolden Rett Syndrome Trial Dosing Q4 2025

- Rein Therapeutics received **European Medicines Agency (EMA) authorization** to initiate its Phase 2 "RENEW" clinical trial for **LTI-03** in idiopathic pulmonary fibrosis (IPF).  **[822370_10b9f57a3f694b17ab45ba11c3c6f0b2_1]** 
- The authorization covers clinical trial sites in **Germany and Poland**, building on previous regulatory clearance from the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA).  **[822370_10b9f57a3f694b17ab45ba11c3c6f0b2_1]** 
- The RENEW trial is a randomized, double-blind, placebo-controlled Phase 2 study evaluating the safety, tolerability, and efficacy of LTI-03, a first-in-class therapy designed to both reduce lung scarring and promote repair.  **[822370_10b9f57a3f694b17ab45ba11c3c6f0b2_0]** **[822370_10b9f57a3f694b17ab45ba11c3c6f0b2_1]** 
- The study plans to enroll up to **120 patients globally** with a treatment duration of **24 weeks**.  **[822370_10b9f57a3f694b17ab45ba11c3c6f0b2_1]**

Rein Therapeutics Receives European Approval to Initiate Phase 2 LTI-03 Trial

- Ascendis Pharma A/S has submitted a **Marketing Authorisation Application (MAA)** to the European Medicines Agency (EMA) for **TransCon CNP (navepegritide)**, a treatment for children with achondroplasia.  **[822370_d0a55749497a4daa86fe2ff4400b6198_0]** 
- The MAA is based on data from **three randomized, double-blind, placebo-controlled clinical trials** and up to **three years of open-label extension data**, showing TransCon CNP was generally well tolerated.  **[822370_d0a55749497a4daa86fe2ff4400b6198_0]** 
- In the United States, TransCon CNP is under **priority review by the U.S. Food and Drug Administration (FDA)**, with a Prescription Drug User Fee Act (PDUFA) target date of **November 30, 2025**.  **[822370_d0a55749497a4daa86fe2ff4400b6198_0]**

Ascendis Pharma Submits MAA to EMA for TransCon CNP

- MaaT Pharma announced **positive results** from the second interim safety analysis by the Data Safety Monitoring Board (DSMB) for its **Phase 2b clinical trial** evaluating MaaT033 in patients receiving allo-GCSH.  **[822370_29585495b2314286a8874e9a8e736269_0]** 
- The DSMB found **no unfavorable safety signals or excess mortality** attributable to MaaT033 among the **first 120 patients** and recommended the **continuation of the study as planned**.  **[822370_29585495b2314286a8874e9a8e736269_0]** 
- The PHOEBUS trial, which is the largest randomized controlled trial evaluating a microbiota-based therapy in oncology globally, is ongoing and aims to enroll **387 patients** across up to **60 sites**.  **[822370_29585495b2314286a8874e9a8e736269_0]** **[822370_29585495b2314286a8874e9a8e736269_1]** 
- MaaT033, an oral Microbiome Ecosystem Therapy, is designed for outpatient use and targets a potential market estimated at **€500 million** (EU5, US) for approximately **6,000 patients per year**.  **[822370_29585495b2314286a8874e9a8e736269_1]**

MaaT Pharma Announces Positive Interim Safety Analysis for MaaT033 Phase 2b Clinical Trial

- Wugen, Inc. announced the closing of **$115 million** in equity financing.  **[822370_a4c29c37db5c410ba17761b5eb69adbf_0]** 
- The financing proceeds will fund the ongoing pivotal **T-RRex trial** for **WU-CART-007** in relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL).  **[822370_a4c29c37db5c410ba17761b5eb69adbf_0]** **[822370_a4c29c37db5c410ba17761b5eb69adbf_1]** 
- **WU-CART-007**, an allogeneic CAR-T cell therapy, achieved a **91% overall response rate** in a global Phase 1/2 study.  **[822370_a4c29c37db5c410ba17761b5eb69adbf_0]** 
- Wugen targets a Biologics License Application (BLA) filing for **WU-CART-007** in **2027**.  **[822370_a4c29c37db5c410ba17761b5eb69adbf_0]** **[822370_a4c29c37db5c410ba17761b5eb69adbf_1]**

Emmaus Life Sciences (EMMA)