On April 30, 2025 Genocury Biotech reported groundbreaking clinical data from its noval in vivo CD19 CAR-T therapy in relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL), a notoriously aggressive blood cancer (Press release, Shenzhen Genocury Biotech, APR 30, 2025, View Source [SID1234652399]). In an investigator-initiated trial (IIT) led by the Hematology Department at Tongji Hospital, a heavily pretreated relapsed/refractory (R/R) patient with advanced DLBCL achieved complete remission (CR) after 1 month of the in vivo CAR-T treatment, with durable response sustained over three months. Notably, the treatment eliminated the need for lymphodepletion, a standard yet toxic preconditioning step in traditional CAR-T protocols.

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Clinical Breakthrough: Redefining Safety, Efficacy, and Speed

Key trial findings:

1. Rapid and Deep Response:

A relapsed DLBCL male patient achieved complete hematological remission (CR) within 28 days following a single dose of Genocury’s CD19 in vivo CAR-T without lymphodepletion. Flow cytometric analysis revealed significant CAR-T cell expansion kinetics, with sustained therapeutic efficacy confirmed through 90-day follow-up monitoring.

2. Unprecedented Safety Profile:

Unlike conventional CAR-T, which carries a ~50% risk of cytokine release syndrome (CRS) and neurotoxicity, this novel therapeutic approach demonstrated complete absence of:

Cytokine release syndrome (CRS)
Immune effector cell-associated neurotoxicity syndrome (ICANS)
Lymphodepletion-related complications – typically associated with increased infection risk- further underscores the therapy’s safety profile.
Reimagining CAR-T: In Vivo Engineering Breakthroughs

Genocury’s in vivo CAR-T platform overcomes two critical roadblocks in current CAR-T therapy:

Eliminates Ex Vivo Manipulation: Traditional CAR-T requires 3-4 week vein-to-vein timeline by harvesting, modifying, and expanding a patient’s T cells outside the body, and $400K price tag stems from this time-consuming process. Genocury’s proprietary in vivo CAR-T vector delivers CAR payload directly into circulating T cells in vivo, enabling functional CAR-T generation.
Lymphodepletion-Free Protocol: Current protocols require the harsh preconditioning chemotherapy for immune reset – a leading cause of hospitalization. Genocury’s lymphodepletion-free approach reduces treatment-related complications and logistical burdens, as well as enabling significant CAR-T cell expansion.
Statement from Prof. Jia Wei , M.D., Ph.D.

PI (principal investigator), Tongji Hospital Hematology Department (Wuhan)
"In this groundbreaking case, we observed the patient treated with Genocury’s CD19 in vivo CAR-T achieved complete remission through 90-day follow-up – achieved without any lymphodepletion, which fundamentally challenges current cellular therapy dogma" said Dr. Jia Wei, "This therapy combines the benefits of autologous CAR-T with the accessibility of universal therapies, potentially ending the era of unaffordable cancer treatments. This could democratize access to CAR-T globally, we are very excited to advance this paradigm-shifting approach."

On April 30, 2025 Mabwell (688062.SH), an innovation-driven biopharmaceutical company with entire industry chain, reported 6 study results of innovative drugs and platforms in poster format at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting, held from April 25 to 30, 2025 (Press release, Mabwell Biotech, APR 30, 2025, View Source [SID1234652398]).

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Poster Presentation

1. A B7-H3-targeting antibody-drug conjugate, 7MW3711, and PARP inhibitors synergistically potentiate the antitumor activity in B7-H3-positive cancers

Published Abstract Number: 830

Both PARP inhibitors and B7-H3-targeting ADC are feasible therapies to chemotherapy resistant solid tumors. Mabwell combines its self-developed 7MW3711 with PARP inhibitors to explore the synergistic antitumor activity of the ADC+PARPi combination strategy in preclinical solid tumor models.

In this study, the synergistic antitumor activity shown by 7MW3711 in combination with PARPi suggests that it is a promising strategy to combine DNA damage repair inhibitor and B7-H3-targeting ADC for the treatment of B7-H3-positive solid tumors. The data provide evidence for the potential utility of 7MW3711 combination with PARPi for treatment of B7-H3-expressing tumors and support the rationale for further clinical application.

2. Design and synthesis of the novel camptothecin analog MF6 for application into site-specific antibody-drug conjugate

Published Abstract Number: 5733

This study preliminarily validated the antitumor activity and safety of MF6, a novel payload based on the Mtoxin platform, in multiple in vivo and in vitro models. The experimental results demonstrated that MF6 possessed favorable tumor-killing activity and maintains significant efficacy in multidrug-resistant models resistant to DXd. ADCs constructed using the clinically validated site-specific conjugation technology IDDC and our novel payload MF6 demonstrated good uniformity and stability, and exhibited significant antitumor efficacy in multiple CDX models. Additionally, these ADCs show a potent bystander killing effect enabling the killing of nearby tumor cells and thereby further enhancing the antitumor efficacy. ADCs synthesized with MF6 have excellent serum and plasma stability and pharmacokinetic properties, providing strong support for future clinical application.

3. MW-C01/C02, novel CLDN1-targeting antibody-drug conjugates, demonstrate compelling anti-tumor efficacy and favorable safety profiles in preclinical studies

Published Abstract Number: 1573

MW-C01/C02 are novel CLDN1-targeting ADCs, developed based on our own ADC site-specific conjugation technology platform IDDC. Claudins localize to tight junctions in healthy tissues, while their overexpression in solid tumors leads to aberrant exposure outside of these junctions, making them attractive targets for ADC therapies. Studies have shown that high expression of CLDN1 is associated with tumor proliferation, invasion, metastasis, and poor prognosis. MW-C01/C02 exhibit robust binding, rapid internalization, and potent cytotoxicity in CLDN1-positive cancer cell lines. MW-C01/C02 demonstrate potent anti-tumor activity in both preclinical CDX and PDX models and show good PK and safety profiles in primates.

4. 2MW7061, a novel LILRB4xCD3 bispecific T-cell engager targeting monocytic acute myeloid leukemia

Published Abstract Number: 2116

2MW7061 (LILRB4xCD3) is a bispecific T-cell engager (TCE) targeting LILRB4 developed based on Mabwell’s TCE platform. With its unique structural design and mechanism of action, 2MW7061 exhibits minimal binding to T cells while demonstrating potent cytotoxicity against tumor cells, thereby significantly improving its safety margin without compromising efficacy. In preclinical models of AML (acute myeloid leukemia), 2MW7061 showed strong anti-tumor activity. Non-primate toxicology studies indicate a favorable safety profile, highlighting its therapeutic potential for treating AML patients.

5. An innovative T cell engager platform with optimized CD3 affinity and formats for targeting hematologic and solid tumors

Published Abstract Number: 2866

As a promising cancer therapeutic strategy, T‑cell engagers (TCEs) simultaneously bind to CD3 on T cells and tumor-associated antigens (TAAs) on cancer cells, facilitating the formation of an immunological synapse that activates T cells and directs their cytotoxic activity toward tumor cells. Clinical data have robustly demonstrated the efficacy of TCEs in hematologic malignancies; however, their clinical benefits in solid tumors remain to be fully validated. As agonists, TCEs present significant developmental challenges that require a delicate balance between efficacy and safety. In response, Mabwell has established an innovative TCE platform that meticulously optimizes various parameters—including CD3 affinity, TAA selection, and bispecific antibody format—to effectively widen the therapeutic window and maximize clinical outcomes.

6. 7MW4911, a novel cadherin 17-targeting ADC, demonstrates potent efficacy in preclinical models of gastrointestinal cancers

Published Abstract Number: 5466

7MW4911 is a novel ADC developed by Mabwell targeting CDH17, a membrane protein highly expressed in gastrointestinal cancers with limited normal tissue distribution, offering a promising therapeutic strategy for gastrointestinal malignancies. 7MW4911 is composed of a proprietary anti-CDH17 monoclonal antibody, a cleavable linker, and innovative cytotoxic payload Mtoxin, with full intellectual property ownership. In preclinical CDX and PDX models, 7MW4911 demonstrated robust antitumor activity and superior efficacy over MMAE-based ADCs in multidrug resistance models. Pharmacokinetic and safety studies in non-human primates support a favorable profile and a high non-severely toxic dose (HNSTD), enabling clinical advancement. 7MW4911 represents a differentiated and promising ADC candidate for GI cancers, with an IND submission to the NMPA and FDA planned for H2 2025.

On April 30, 2025 Bold Therapeutics Inc., a clinical-stage biopharmaceutical company pioneering novel oncology treatments, reported that its Sr. Director of Preclinical Development, Mark Bazett, PhD, is presenting a late-breaking poster on BOLD-100’s potent neuroprotective properties later today at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 (Press release, Bold Therapeutics, APR 30, 2025, View Source [SID1234652397]).

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Session: Late-Breaking Research: Experimental and Molecular Therapeutics 4

Neuropathy Rates from BOLD-100-001 vs. Historical Controls
Neuropathy Rates from BOLD-100-001 vs. Historical Controls
Bold Therapeutics’ Data in a Model of Cold Allodynia
Bold Therapeutics’ Data in a Model of Cold Allodynia
Date & Time: April 30, 2025, from 9:00 AM to 12:00 PM

Location: Poster Section 51, Poster Board #14

Poster Code: #LB432

Poster Title: "Clinical-stage anticancer agent BOLD-100 demonstrates protective effects against oxaliplatin-induced peripheral neuropathy in an in-vivo rat model"

Addressing a Critical Unmet Need: Oxaliplatin-Induced Peripheral Neuropathy

In Bold Therapeutics’ global Phase 1b/2a trial (BOLD-100-001, NCT04421820), patients with advanced gastrointestinal cancers — including colorectal (mCRC), gastric (GC), biliary tract (BTC), and pancreatic cancer (PDAC) — were treated with BOLD-100 in combination with FOLFOX (5-fluorouracil, oxaliplatin, leucovorin). Across 109 patients in six treatment arms, the study showed encouraging efficacy data, which was previously presented at ESMO (Free ESMO Whitepaper) GI and ASCO (Free ASCO Whitepaper) in 2024.

A surprising finding from this trial was a profound reduction in both the frequency and severity of oxaliplatin-induced peripheral neuropathy (OIPN), a relatively common and debilitating side effect of chemotherapy. In each patient cohort, rates of neuropathy were dramatically lower than those typically seen in historical benchmarks for FOLFOX alone:

These results were supported by feedback from trial investigators, many of whom noted the unexpectedly low incidence of neuropathy in their patients treated with BOLD-100 — particularly those who were heavily pretreated and/or receiving FOLFOX again where one would expect both a high incidence and severity of neuropathy.

To better understand this effect, Bold Therapeutics developed a preclinical rat model of cold allodynia, a common symptom of OIPN. Rats were treated with either high or low doses of oxaliplatin and then given BOLD-100 every three days for 17 days. The results confirmed the clinical findings: rats receiving BOLD-100 showed significantly less neuropathic pain. Once treatment with BOLD-100 stopped, neuropathy symptoms rapidly returned, further supporting its protective effect.

These findings prompted further efforts to elucidate BOLD-100’s neuroprotective mechanism and investigation into whether BOLD-100 can prevent or reduce neuropathy from other neuropathy-inducing agents, such as the taxanes. BOLD-100’s unique ability to both directly impact cancer while simultaneously preventing or reducing OIPN could redefine first-line treatment by helping patients:

Improve overall outcomes through direct anti-cancer activity and completing full chemotherapy cycles.
Stay on therapy longer without dose reductions or early discontinuation.
Experience less pain, tingling, and sensory issues that impact daily life.
Avoid long-term nerve damage that can persist long after treatment ends.
Ongoing Clinical Study and Future Outlook

Bold Therapeutics is currently enrolling FOLFOX-naïve second-line mCRC patients in a multinational randomized clinical study comparing FOLFOX vs. FOLFOX + BOLD-100 in various efficacy, safety, and quality-of-life endpoints. Bold Therapeutics anticipates this trial will further demonstrate BOLD-100’s potential as a transformative therapy in early-line colorectal cancer, biliary tract cancer and other solid tumor indications.

Bold Therapeutics invites investors, strategic partners, clinical investigators and key opinion leaders to connect with the team at AACR (Free AACR Whitepaper) to discuss these latest findings, the company’s ongoing clinical development plans, and potential strategic collaborations.

On April 30, 2025 Rgenta Therapeutics, a clinical-stage biotechnology company pioneering the development of a new class of oral small molecules targeting RNA and RNA regulation for oncology and neurological disorders, reported that preclinical data will be presented on its proprietary RSwitch technology, which enables the fine-tuning of transgene levels in gene and cell therapy applications, at the American Society of Gene And Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, which will be held from May 13 -17th, 2025, in New Orleans, LA (Press release, Rgenta Therapeutics, APR 30, 2025, View Source [SID1234652396]).

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Title: RSwitch Enabled Gene Therapy to Fine Tune Frataxin Expression for the Treatment of Friedrich’s Ataxia
Authors: Samuel A. Hasson, Jon Dempersmier, Mariam Elhawary, Diane Hamann, Ian McLachlan, Chris Yates, Travis Wager, and Simon Xi
Session date and time: Wednesday, May 14, 2025, 5:30 – 7:00 PM CT
Location: Poster Hall I2
Poster ID: AMA251

About RSwitch
RSwitch is a proprietary regulatable gene therapy system that enables oral, small molecule drug control of transgene levels in gene and cell therapy applications. RSwitch encodes a "dimmer" switch that makes the expression of transgene dependent on the administration of an oral small molecule drug that controls the system. Only when the drug is administered is the system activated. Furthermore, the level of gene expression is dependent on how much drug is administered. This precise gene control has the potential to enable fine control of the expression of a therapeutic protein. Rgenta has demonstrated the RSwitch system’s feasibility in vitro and in vivo, achieving dose-dependent expression of reporter transgenes following small molecule administration. RSwitch technology offers versatile control across multiple gene and cell therapy applications, and the company is actively exploring strategic partnerships.

On April 30, 2025 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the Company will participate in the BofA Securities 2025 Healthcare Conference. Company management will participate in a fireside chat on Wednesday, May 14, 2025, at 10:00 a.m. PT / 1:00 p.m. ET / 6:00 p.m. IST (Press release, Jazz Pharmaceuticals, APR 30, 2025, View Source [SID1234652395]).

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An audio webcast of the fireside chat will be available via the Investors section of the Jazz Pharmaceuticals website at View Source A replay of the webcast will be archived on the website for 30 days.