Treating sickle cell earlier
Preliminary data from two ongoing pediatric trials suggest that the gene therapy exagamglogene autotemcel (exa-cel) may offer a treatment option for children with beta-thalassemia or sickle cell disease. Although exa-cel is already approved for patients 12 and older, researchers say the therapy may have particular value when used earlier in life, before long-term organ damage develops.
In the two studies, which enrolled children aged 5 to 11, all participants with enough follow-up met their primary endpoints. Among the 13 children treated for beta-thalassemia in the CLIMB THAL-141 trial, six have been followed long enough to assess whether they could maintain transfusion independence for 12 months. All six achieved this, and increases in hemoglobin and fetal hemoglobin production were also observed. In the CLIMB SCD-151 trial, 4 of the 11 children treated for sickle cell disease met the endpoint of remaining free from severe vaso-occlusive crises for a full year. None have experienced a crisis after receiving the therapy, and hemoglobin levels generally returned to normal by month six.
Researchers note that the treatment process has shown a safety profile consistent with experiences in older patients, although one child in the beta-thalassemia study died from severe veno-occlusive disease related to busulfan. This complication is a known risk of conditioning regimens, particularly in younger patients.
Both trials, sponsored by Vertex Pharmaceuticals and CRISPR Therapeutics, will continue to enroll and monitor participants to better understand long-term safety and durability. Investigators also anticipate exploring the therapy in even younger children in future studies.
Funding for breast cancer therapy
Pan Cancer T has secured EUR 10 million in new financing to support the first clinical study of its lead TCR-T cell therapy, PCT1:CO-STIM, in women with triple-negative breast cancer. The funding combines EUR 5 million from existing investors with a EUR 5 million Innovation Credit loan from the Dutch Ministry of Economic Affairs. The company plans to begin treating patients at cancer centers in the Netherlands after submitting a regulatory filing to the EMA in 2026.
PCT1:CO-STIM is an autologous T cell therapy engineered with a T-cell receptor specific for ROPN1, a tumor-restricted target found in more than 90 percent of cases. The therapy also incorporates a co-stimulatory technology intended to improve T cell activity in the solid tumour microenvironment. The upcoming first-in-human study will evaluate safety, tolerability, and early signs of efficacy in a patient population with limited treatment options and poor overall survival following metastatic disease.
New approval for BMS
The FDA has approved Bristol Myers Squibb’s CAR T cell therapy Breyanzi (lisocabtagene maraleucel) for adults with relapsed or refractory marginal zone lymphoma who have received at least two prior systemic treatments. With this decision, Breyanzi becomes the only CAR T therapy authorized in five cancer types and the only CAR T option available for this patient population.
The approval is supported by data from the marginal zone lymphoma cohort of the TRANSCEND FL study, an open-label, multicenter, single-arm trial. Among 66 evaluable patients treated in the third-line and later setting, the overall response rate was 95.5 percent, including a 62.1 percent complete response rate. The median duration of response was not reached at the time of analysis, with more than 90 percent of responders maintaining their response at 24 months.
The ballroom way
Cellipont Bioservices in Texas has expanded its manufacturing capabilities by completing a ballroom-style cleanroom equipped with a 50-l single-use bioreactor. The new facility is intended to support cGMP-compliant production for exosome, induced pluripotent stem cell, and mesenchymal stem cell therapies, addressing rising demand for scalable allogeneic manufacturing.
The new 50-l system is designed to provide a bridge from early development to commercial-scale production by enabling larger batch volumes, improving process scalability, and supporting efforts to achieve consistent product quality. According to the company, these capabilities are expected to facilitate more efficient manufacturing timelines for clients advancing allogeneic cell therapies.
Cellipont describes the expansion as part of its broader strategy to increase capacity for next-generation cell therapy programs.
Supporting cell therapy manufacturing
PBS Biotech is launching the MiniPRO, a multi-parallel version of its Vertical-Wheel bioreactor platform designed to support high-throughput process development for cell therapy manufacturing. The system allows researchers to run as many as 24 independent small-scale cultures within an automated, closed environment that offers real-time control and monitoring of temperature, agitation, oxygenation, pH, and perfusion-based media exchange.
MiniPRO is based on the company’s established Vertical-Wheel technology, which is also used in its GMP-ready bioreactors.
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