On October 7, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported that the results of CT071 (an autologous CAR T-cell product targeting GPRC5D) for the treatment of relapsed/refractory multiple myeloma (R/R MM) in an investigator-initiated trial (NCT05838131) have been published in The Lancet Haematology (Press release, Carsgen Therapeutics, OCT 7, 2025, View Source [SID1234656494]). The article was titled "GPRC5D-targeted CAR T-cell therapy (CT071) in patients with relapsed or refractory multiple myeloma: a first-in-human, single-centre, single-arm, phase 1 trial".

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This trial aimed to assess the safety, preliminary activity of CT071 in R/R MM. 20 patients received CT071 infusion. Patients had received a median of 5 prior lines of therapy (IQR 3.0-6.5); 19 (95%) were double-class refractory, 13 (65%) were triple-class refractory, 5 (25%) were penta-drug refractory, 10 (50%) had received autologous stem cell transplantation (ASCT), and 5 (25%) had relapsed after CAR T-cell therapies targeting BCMA or BCMA/CD19. Four (20%) patients had extramedullary disease (EMD), 14 (70%) had ≥1 high-risk cytogenetics and 19 (95%) had a Revised International Staging System (R-ISS) 2 or 3 disease at baseline.

No dose-limiting toxicities (DLTs) were observed. The recommended phase 2 dose was determined at 0.1×106 CAR T cells/kg. Cytokine release syndrome occurred in 12 patients (60%), all graded as 1 or 2. No Grade ≥3 cytokine release syndrome (CRS) occurred. One patient (5%) experienced Grade 3 immune effector cell-associated neurotoxicity syndrome (ICANS). No treatment-related deaths occurred.

With a median follow-up of 10.71 months (IQR 6.13-12.02), the objective response rate (ORR) was 100% (95%CI, 83.2-100). 10 (50%) patients achieved stringent complete response (sCR), 4 (20%) had very good partial response (VGPR) and 6 (30%) had partial response (PR). One patient with a large EMD (125 mm×99 mm at baseline) achieved a 67.6% reduction at month 10 with ongoing PR. All 5 patients previously treated with an anti-BCMA CAR T (n=1) or anti-BCMA/CD19 CAR T (n=4) responded; 2 achieved PR, 1 achieved VGPR and 2 achieved sCR. 18 of 20 (90%) evaluable patients achieved MRD negativity at 10−6 including all 10 with CR or sCR. Median time to MRD negativity was 29 days (IQR 29-29). Median duration of response (DoR), progression-free survival (PFS) and overall survival (OS) were not reached. The data cutoff for all analyses was December 9, 2024.

About CT071
CT071 is a CAR T-cell therapy candidate developed utilizing the proprietary CARcelerate platform targeting GPRC5D for the treatment of relapsed/refractory multiple myeloma (R/R MM) or plasma cell leukemia (PCL). Initiated trials include an investigator-initiated trial for R/R MM or PCL in China (NCT05838131), and an investigator-initiated trial for newly diagnosed multiple myeloma (NDMM) in China (NCT06407947).

On October 7, 2025 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a clinical-stage diabetes and obesity company, reported the pricing of its previously announced underwritten public offering consisting of (i) 11,195,121 shares of its common stock and accompanying warrants to purchase an aggregate of 11,195,121 shares of common stock (or pre-funded warrants in lieu thereof) and (ii) in lieu of common stock, to certain investors, pre-funded warrants to purchase an aggregate of up to 1,000,000 shares of its common stock and accompanying warrants to purchase an aggregate of up to 1,000,000 shares of common stock (or pre-funded warrants in lieu thereof), at an exercise price of $0.0001 per pre-funded warrant (Press release, Biomea Fusion, OCT 7, 2025, View Source [SID1234656479]). In addition, Biomea has granted the underwriters a 30-day option to purchase up to an additional 1,829,268 shares of common stock and/or warrants to purchase 1,829,268 shares of common stock at the public offering price, less underwriting discounts and commissions. The common stock and pre-funded warrants are being sold in combination with an accompanying warrant to purchase one share of common stock (or a pre-funded warrant in lieu thereof) issued for each share of common stock or pre-funded warrant sold. The accompanying warrant has an exercise price of $2.50 per share, is immediately exercisable from the date of issuance and will expire three years from the date of issuance. The combined offering price of each share of common stock and accompanying common stock warrant is $2.05. The combined offering price of each pre-funded warrant and accompanying common stock warrant is $2.0499.

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All of the shares, pre-funded warrants and accompanying common stock warrants in the offering are being sold by Biomea. The gross proceeds to Biomea from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be approximately $25.0 million, excluding any exercise of the underwriters’ option to purchase additional shares and/or warrants. The offering is expected to close on October 8, 2025, subject to the satisfaction of customary closing conditions.

Jefferies is acting as sole book-running manager for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering.

The shares of common stock, pre-funded warrants and common stock warrants and shares of common stock issuable upon the exercise of the pre-funded warrants and common stock warrants are being offered by Biomea pursuant to an effective shelf registration statement on Form S-3 (File No. 333-289262), that was previously filed with the U.S. Securities and Exchange Commission ("SEC") on August 5, 2025 and declared effective on August 15, 2025. A preliminary prospectus supplement relating to and describing the terms of the offering was filed with the SEC on October 6, 2025, and is available for free on the SEC’s website located at View Source The final prospectus supplement and accompanying prospectus relating to and describing the terms of the offering will be filed with the SEC and will be available for free on the SEC’s website located at View Source

Copies of the final prospectus supplement and the accompanying prospectus relating to the offering, when available, may also be obtained from: Jefferies LLC by mail at Attn: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388, or by email at [email protected]; or by accessing the SEC’s website at www.sec.gov.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On October 7, 2025 Cyteph Pty Ltd, a biotechnology company developing novel immunotherapies for difficult-to-treat cancers, reported that it has successfully completed recruitment for its first-in-human Phase I clinical trial of CYT-101 (Press release, Cyteph, OCT 7, 2025, View Source [SID1234656495]).

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CYT-101 is a novel off-the-shelf, HLA-matched CMV-specific T cell therapy for the treatment of glioblastoma multiforme (GBM) and other solid cancers. GBM is an aggressive brain cancer and one of the deadliest solid cancers in adults, with limited treatment options available.

The unique advantage of targeting CMV antigens on GBM cells is that they are detected on tumour cells while not found in surrounding healthy tissue. This characteristic allows for a highly targeted approach. By harnessing the power of allogeneic CMV-specific T cell therapy, CYT-101 has the potential to offer improved outcomes for GBM patients.

The Phase I study is being conducted in collaboration with Briz Brain & Spine and Newro Foundation and is evaluating the safety, tolerability, and preliminary signals of efficacy of CYT-101 in patients with recurrent GBM.

"We are pleased to have completed enrolment, reflecting the strong demand from clinical collaborators and patients for innovative treatment options for GBM," said Professor Rajiv Khanna, Chief Scientific Officer and Founder of Cyteph. "This milestone marks an important step forward in our mission to bring transformative therapies to patients with few existing options."

The Phase I trial follows participants through dose-escalation cohorts, with key readouts expected at the end of Q4 2025.

Results from this study will inform the design of subsequent clinical trials and support the continued development of CYT-101 as a novel treatment approach for glioblastoma and other high-unmet-need solid cancers.

"Completing recruitment is a critical milestone for any clinical program, and it underscores the dedication of our team, and the patients who make this research possible," said Professor David Walker, neurosurgeon and spinal surgeon at Briz Brain & Spine and lead clinical investigator for the CYT-101 clinical trial.

The CYT-101 Phase I clinical trial is funded through Australia’s national biotech incubator CUREator.

Cyteph is a spin-out biotechnology company from QIMR Berghofer, a leading medical research institute based in Brisbane, Australia.

On October 7, 2025 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, reported that findings highlighting the strong potential of circular single-stranded DNA (CssDNA) as a universal, efficient non-viral template for gene therapy, along with a comprehensive study of TALE base editors (TALEB) off-targets in the nuclear genome, will be presented at the European Society of Cell and Gene Therapy (ESGCT) annual congress, that will take place on October 7-10, 2025, in Sevilla, Spain (Press release, Cellectis, OCT 7, 2025, View Source [SID1234656480]).

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Poster presentation:

Title: Circularization of Single-Stranded DNA Donor Template Unleashes the Power of Non-Viral Gene Delivery for Long-Term HSCs editing

Presenter: Julien Valton, Ph.D., Vice President Gene Therapy at Cellectis

Date/Time: Wednesday, October 8, 2025 from 2:00 p.m. to 3:30 p.m. CET

Poster number: P0439

Over the past decade, non-viral DNA template delivery has been used with engineered nucleases to target single-stranded DNA sequences in hematopoietic stem and progenitor cells (HSPCs).

While developed for gene therapy purposes, so far this method has been restricting to gene corrections. To expand this scope, Cellectis developed an editing process using its gene editing technology and kilobase-long circular single-stranded DNA donor templates.

Research data show that:

· CssDNA editing process achieved high gene insertion frequency in viable HSPCs.

· CssDNA-edited HSPCs show a higher propensity to engraft and maintain gene edits in a murine model than adeno-associated viruses (AAV)-edited HSPCs.

These data highlight the strong potential of CssDNA as a universal and efficient non-viral DNA template for gene therapy applications.

The research was also presented as an oral presentation at the Homology-Directed Repair: The Path Forward Workshop in Sevilla on October 6, 2025.

Poster presentation:

Title: Comprehensive analysis of TALEB off-target editing

Presenter: Maria Feola, Ph.D., Senior Scientist, Team Leader Gene Editing at Cellectis

Date/Time: Thursday, October 9, 2025 from 2:00 p.m. to 3:30 p.m. CET

Poster number: P0506

TALE base editors (TALEB) are fusions of a transcription activator-like effector domain (TALE), split-DddA deaminase halves, and an uracil glycosylase inhibitor (UGI).

These recent additions to the genome editing toolbox can directly edit double strand DNA, converting a cytosine (C) to a thymine (T) through the formation of an uracil (U) intermediate without the need of DNA break. Base editing has great potential in therapeutic applications. However, being able to avoid potential off-target effects is key toward this goal.

To evaluate TALEB safety, Cellectis combined advanced bioinformatic predictions with multiple experimental approaches to investigate potential off-target effects in the nuclear genome of primary T cells.

The study found no evidence of biases towards off-site C-to-T editing at sites flanked by CTCF binding sites, a key DNA-binding protein that regulates genome organization and gene expression at genome wide level.

These results provide a strong framework for the safe development of TALEB in therapeutic cell engineering, supporting their potential for future nuclear and mitochondrial applications.

On October 7, 2025 Precision NeuroMed (PNM), a clinical-stage biotechnology company pioneering advanced drug delivery technologies for central nervous system (CNS) diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its investigational therapy for glioblastoma (GBM) (Press release, Precision NeuroMed, OCT 7, 2025, View Source [SID1234656496]).

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PNM is reintroducing cintredekin besudotox (IL13-PE38QQR), a potent cytotoxic protein designed to target and kill cells that express the IL-13 alpha-2 receptor (IL13aR), uniquely present on tumor cells such as glioblastoma. The protein’s selective expression allows it to precisely attack cancer cells while minimizing damage to normal brain tissue.

Despite advancements in understanding the genetics of glioblastoma and identifying novel drug targets, the blood–brain barrier (BBB) remains a significant obstacle, severely limiting the effectiveness of many potential therapies delivered through the bloodstream. PNM’s platform uses convection-enhanced delivery (CED) to bypass the BBB and enable direct, targeted administration of nanoparticles, including proteins, liposomes, and gene therapy at therapeutic concentrations to the brain.

"Receiving Orphan Drug Designation is an important milestone for Precision NeuroMed as we advance our mission to transform treatment for patients with glioblastoma, one of the most aggressive and devastating brain cancers," said Sandeep Kunwar, MD, CEO and Co-Founder, Precision NeuroMed. "By combining innovative drugs with our next-generation delivery system, we hope to dramatically improve outcomes where few effective options currently exist."

Each year, more than 12,000 individuals in the United States are expected to succumb to glioblastoma. The five-year survival rate for patients is just 5%, with an average life expectancy of 12 to 18 months following diagnosis. Standard treatment consists of maximal tumor resection followed by radiation therapy with temozolomide with no major innovations since the approval of temozolomide for glioblastoma in 2005.

PNM is developing a personalized approach that targets both molecular and regional aspects of tumor cells within the brain. By improving drug delivery at the site of disease, PNM aims to extend survival and enhance quality of life for patients with glioblastoma in a meaningful way.

Orphan Drug Designation from the U.S. Food and Drug Administration is reserved for therapies intended to treat, diagnose, or prevent rare diseases affecting fewer than 200,000 people in the United States. This designation provides development benefits — such as tax credits for eligible clinical research and exemption from applicable FDA user fees — and, upon approval for the designated indication, confers seven years of market exclusivity.