.png)
Highlights & Summary
Intro
The new year is already off to a busy start regarding clinical trial progresses, financial and personnel updates, and regulatory approvals throughout the Next Gen Therapeutics world. Cell therapy in particular features exciting news regarding innovations in manufacturing.
Happy Reading!
Cell Therapy
Gene Therapy
- Beacon Granted FDA RMAT Designation for XLRP Asset | Regulatory
- GeneCraft’s NSCLC Asset Cleared for Clinical Testing in South Korea | Clinical Trial
- Coave Hauls in ~$33M in Series A | Financing
- iECURE Reports Complete Response in First Infant Dosed with ECUR-506 | Clinical Trial
Oligo Therapy
Antibody-Drug Conjugates
- ENHERTU Approved in the US for HER2-low or HER2-ultralow metastatic breast cancer patients | Regulatory
Cell Therapy
A2 Biotherapeutics, a company developing logic-gated cell therapies for cancer, announced the closing of an $80 million Series C financing round. The funds will be used to support the clinical development of its CAR-T cell therapies based on the Tmod™ platform technology. A2 Bio is currently conducting two clinical studies, EVEREST-1 and EVEREST-2, evaluating its CAR-T cell therapies for various solid tumors. The company's Tmod™ platform incorporates two receptors, an activator and a blocker, to precisely target tumor cells while protecting normal cells.
Phase 1 Initiated for EsoBiotec's BCMA-director in vivo CAR-T for Multiple Myeloma | Clinical Trials
EsoBiotec announced that the first patient has been dosed in a clinical trial of ESO-T01, an in vivo BCMA-directed CAR-T therapy for relapsed/refractory multiple myeloma. ESO-T01 uses a lentiviral vector platform to reprogram patients' T-cells, offering a potentially less expensive and more accessible treatment option compared to traditional ex vivo CAR-T therapies. The trial is a phase 1 dose escalation study and will enroll up to 24 patients. The company anticipates initial clinical data will be available in the second half of 2025.
CellFE Launches T-REST Transfection Kit to Improve CAR-T Manufacturing Efficiency | Manufacturing
CellFE, a company specializing in non-viral gene editing, has introduced its T-Rest Kit, a novel transfection media designed for resting (quiescent) T cell workflows. This product allows for gene editing in quiescent T cells, potentially leading to more effective, higher yield CAR-T cell manufacturing workflows. The T-Rest Kit preserves the stem cell memory of resting T cells, which is critical for developing CAR-T cell treatments with improved durability. Additionally, gene editing in non-dividing cells may reduce the risk of chromosomal abnormalities. This new approach may offer cell therapy manufacturers a new avenue for enhancing the production of CAR-T cell therapies.
FDA Issues Complete Response Letter for Atara's Tabelecleucel in EBV+ PTLD | Regulatory
Atara Biotherapeutics received a Complete Response Letter (CRL) from the FDA regarding its application for tabelecleucel (tab-cel), an EBV-specific T-cell immunotherapy for EBV+ PTLD. The CRL was related to GMP compliance issues at a third-party manufacturer, not to safety or efficacy concerns with the asset itself. Atara is working to address the issues and resubmit the application. If approved, tab-cel would be the first approved therapy in the US for EBV+ PTLD, a rare complication of organ transplantation.
Astraveus Achieves CAR-T Cell Production Using its Microfluidic Lakhesys Platform | Manufacturing
Astraveus has successfully produced CAR-T cells using a fully automated, end-to-end microfluidic system, marking a significant milestone for the company. The company's Lakhesys Benchtop Cell Factory™ integrates the entire CAR-T production process into a compact, benchtop device, potentially reducing manufacturing costs and increasing throughput. "With a production time of only 26 hours, we have also demonstrated that our bead-free microfluidic selection technology enables fast CAR-T cell manufacturing," says CEO Jérémie Laurent. By streamlining and miniaturizing the manufacturing process, this approach could significantly reduce the cost and complexity of cell therapy production.
Gene Therapy
Beacon Granted FDA RMAT Designation for XLRP Asset | Regulatory
Beacon Therapeutics announced that the FDA granted RMAT designation to its gene therapy, laru-zova, for treating X-linked retinitis pigmentosa (XLRP), based on positive Phase 2 trial data. Laru-zova also holds FDA Fast Track, EMA PRIME, and UK MHRA ILAP designations, with ongoing studies showing promising results in improving visual function in XLRP patients. Several other companies are developing similar treatments, with MeiraGTx’s Janssen-partnered AAV5-RPGR in Ph3 testing.
GeneCraft’s NSCLC Asset Cleared for Clinical Testing in South Korea | Clinical Trial
GeneCraft's RX001, a novel AAV-based therapy for non-small cell lung cancer (NSCLC), has received IND clearance from South Korea's Ministry of Food and Drug Safety. The therapy targets K-RAS mutant NSCLC and aims to minimize side effects by selectively eliminating cancer cells while sparing normal cells. ProBio, a key partner in the development, provided plasmid and AAV research, manufacturing, and process support for the asset. Few AAV gene therapies are focused on cancer indications, with Siren Biotechnology being another notable company in this space.
REGENXBIO and Nippon Shinyaku Partner on Asset Commercialization | Partnership
REGENXBIO and Nippon Shinyaku have entered a strategic partnership to develop and commercialize RGX-121 for Mucopolysaccharidosis II (MPS II) and RGX-111 for MPS I. REGENXBIO will receive $110 million upfront and up to $700 million in milestone payments, along with royalties from sales in the U.S. and Asia. The collaboration will leverage REGENXBIO's gene therapy expertise and Nippon Shinyaku's capabilities in rare disease commercialization, with RGX-121 potentially receiving FDA approval as early as late 2025.
Coave Hauls in ~$33M in Series A | Financing
Coave Therapeutics has raised €32 million ($33 million) in Series A financing, co-led by Novo Holdings and Bpifrance, with participation from other investors. The funding will support the advancement of Coave's ALIGATER™ platform, which improves the delivery of genetic payloads to extra-hepatic tissues with enhanced specificity, efficiency, and safety. The company plans to use the financing to advance its preclinical assets, focusing on CNS, neuromuscular, and eye diseases, with plans to begin CTA/IND-enabling studies in 2026.
iECURE Reports Complete Response in First Infant Dosed with ECUR-506 | Clinical Trial
iECURE's ECUR-506, a dual-AAV gene therapy for neonatal onset ornithine transcarbamylase (OTC) deficiency, has shown a complete clinical response in the first infant dosed in a Phase 1/2 trial. The treatment, which utilizes two AAV vectors carrying different payloads, was well tolerated and led to significant clinical improvements, including discontinuation of ammonia scavenger medication and normalization of ammonia levels. This rare dual-AAV approach may represent a breakthrough in treating severe, early-onset genetic liver disorders like OTC deficiency, potentially eliminating the need for liver transplantation.
Oligo
Atalanta Therapeutics Secures $97 Million to Advance CNS-Targeted RNAi Therapies | Financing
Atalanta Therapeutics has closed a $97 million Series B financing to advance two RNAi therapies for CNS diseases to clinical trials. The funding will support Phase 1 clinical trials for KCNT1-related epilepsy and Huntington's disease. Atalanta's platform uses divalent small interfering RNA (di-siRNA) for durable, selective gene silencing throughout the brain and spinal cord. Preclinical studies have shown promising results for both candidates, with ATL-201 reducing seizures in KCNT1-related epilepsy models and ATL-101 producing strong HTT gene silencing in Huntington's disease models. The company plans to submit IND applications for both programs in 2025. The financing was co-led by EQT Life Sciences and Sanofi Ventures, with participation from several other investors.
FDA Lifts Clinical Hold on Amylyx Pharmaceuticals' ALS Drug AMX0114 Trial | Regulatory
Amylyx Pharmaceuticals announced that the FDA has lifted the clinical hold on the Phase 1 trial of AMX0114 for ALS treatment. AMX0114 is an investigational antisense oligonucleotide targeting calpain-2, a protein involved in axonal degeneration. The LUMINA trial, a multicenter, randomized, placebo-controlled study, will evaluate AMX0114's safety, tolerability, and biological activity in approximately 48 ALS patients. The trial will assess changes in calpain-2 levels, neurofilament light (NfL) levels, and other ALS biomarkers. Amylyx expects to initiate the trial in Canada in early 2025 and is now working to open U.S. sites for screening, enrollment, and dosing. The company anticipates early cohort data from LUMINA in 2025.
Eli Lilly Opens Innovative Research Center in Boston Seaport | Commercial
Eli Lilly and Company has opened the Lilly Seaport Innovation Center (LSC) in the Boston Seaport. The 346,000 square foot facility will accommodate approximately 500 Lilly scientists and researchers, plus 200 people from Lilly Gateway Labs companies. LSC will serve as the central hub for Lilly's genetic medicines efforts and house the first East Coast Lilly Gateway Labs. The center is dedicated to advancing Lilly's work in RNA and DNA-based therapies and discovering new drug targets for various diseases. LSC expands Lilly's presence in the Boston area and aims to foster collaboration with leading institutions and new talent. The facility includes laboratories, office space, and state-of-the-art equipment for research and development.
Ethris Reports Promising Phase 1 Results for mRNA Asthma Treatment ETH47 | Clinical Trial
Ethris has presented positive topline data from its Phase 1 clinical trial of ETH47, an mRNA therapeutic candidate for uncontrolled asthma. ETH47 encodes interferon lambda (IFNλ) and is designed to address the upstream trigger of asthma exacerbations. The trial demonstrated that ETH47 was generally safe and well-tolerated, with no serious adverse events reported. The study showed dose-dependent production of IFNλ in nasal lining fluid and activation of antiviral interferon-stimulated genes. Based on these results, Ethris has filed a Clinical Trial Application for a Phase 2a rhinovirus challenge study in asthma patients, planned to begin in Q2 2025. ETH47 utilizes Ethris' proprietary SNIM® RNA and SNaP® LNP platforms for local administration to the respiratory tract.
PepGen Provides Clinical Updates on PGN-EDO51 for Duchenne Muscular Dystrophy | Clinical Trial
PepGen provided updates on its CONNECT clinical program investigating PGN-EDO51 for Duchenne muscular dystrophy (DMD). The CONNECT1 Phase 2 trial has fully enrolled its 10 mg/kg cohort (n=4) and continues dosing in the 5 mg/kg cohort (n=3). Two participants in the 10 mg/kg cohort, previously reported with asymptomatic hypomagnesemia, have returned to baseline magnesium levels with oral supplementation. Dosing for one participant was paused due to reduced estimated glomerular filtration rate (eGFR), which is now improving. Health Canada has allowed continued dosing at current levels but requested additional information before further dose escalation or enrollment. The company is working with the FDA to address questions regarding its CONNECT2 trial, which is currently on clinical hold in the US.
ADCs
Daiichi Sankyo and AstraZeneca’s DATROWAY FDA approved for HR+, HER2- Breast Cancer | Regulatory
Daiichi Sankyo and AstraZeneca announced that their TROP-2 directed antibody drug conjugate DATROWAY has been approved by the FDA for use in metastatic, HR-positive, HER2-negative breast cancer patients. The approval is tied to significant reduction of risk of death or disease progression as demonstrated in TROPION-Breast01. Other regulatory submissions for DATROWAY, such as in the EU and China, are under review. This marks the eighth new medicine that AstraZeneca has set to deliver within the next 5 years.
ENHERTU Approved in the US for HER2-low or HER2-ultralow metastatic breast cancer patients | Regulatory
Daiichi Sankyo and AstraZeneca’s HER-2 directed DXd antibody drug conjugate has been granted FDA approval as the first HER2-directed therapy in metastatic breast cancer patients who are HER2-low or HER2-ultralow statuses. The approval comes after findings in the DESTINY-Breast06 phase III trial, which indicated that ENHERTU resulted in a longer progression-free survival than chemotherapy in HER2-low or HER2-ultralow metastatic breast cancer patients who had already received endocrine-based therapy. Other regulatory bodies across geographies, including Japan and in the EU, are reviewing these findings.
FDA Grants Breakthrough Device Designation to GSK's ADC for Osteosarcoma Patients | Regulatory
GSK announced that the FDA granted Breakthrough Device Designation (BDD) for their GSK’227 antibody drug conjugate for some adult patients with relapsed or refractory osteosarcoma. The ARTEMIS-002 study, a phase II clinical trial, as presented at ASCO’s 2024 Annual meeting, supported this designation through demonstrating positive antitumor activity in pretreated, relapsed or refractory osteosarcoma patients. Osteosarcoma, though a rare disease, accounts for 20-40% of all bone cancers. Relapsed and refractory osteosarcoma patients lack a clear standard of care, creating opportunity for potential innovations such as GSK’227.
Hiroyuki Okuzawa appointed to CEO of Daiichi Sankyo | Personnel
Hiroyuki Okuzawa will transition to Representative Director, President and CEO of Daiichi Sankyo, while current CEO Sunao Manabe will transition to Representative Director and Executive Chairperson, effective April 1 of this year. Dr. Manabe’s leadership was a pinnacle to the growth of antibody drug conjugates in the pharmaceutical industry, for instance, in the commercialization of ENHERTU and DATROWAY.
Synaffix's ADC License to Expand Boehringer Ingelheim Oncology Portfolio | Partnership
Boehringer Ingelheim has joined a partnership with Synaffix, licensing their ADC technology. Synaffix’s clinically validated platform technology support development of ADCs or bispecifics. Boehringer Ingelheim’s subsidiary, NBE Therapeutics, aims to broaden their portfolio with this expansion. Under the agreement, Synaffix will provide access to an undisclosed, proprietary number of targets; Synaffix is eligible to receive milestone payments surpassing $1B. This announcement comes just one day after Synaffix’s announced licensing agreement with Mitsubishi Tanabe, where Synaffix will be responsible for manufacturing critical components related to proprietary technologies while Mitsubishi Tanabe focuses development and commercialization of a specific antibody drug conjugate.
.png)
.png)
