Chinese investors’ interest in cell and gene therapy remains strong
Shi Yingzi ·03/15/2022
China Biotech Fundraising Tracker

By Shi Yingzhi and Chu Minhua

Chinese investors showed strong interest in cell and gene therapy players in February 2022 via financing rounds, continuing a trend seen at the start of the year.   Companies in these areas made up half of the financing rounds disclosed, with cell therapy specialists being the biggest fundraisers.

A total of 21 fundraising deals in drug developers were recorded, significantly lower than the 41 deals in January 2022. The capital raised from the 21 financings amounted to $441 million. The average amount for each financing is $21 million, slightly lower than the $25 million from the previous month.

February saw a distinct lack in high-value financing deals, with none surpassing the $50 million mark. New startup GluBio Pharmaceutical closed the biggest deal, with nearly $50 million raised in a Series A round led by GL Ventures. GluBio specializes in molecular glue degraders and has established three technology platforms for multidimensional protein degradation screening, validating novel targets and designing molecular glue degraders.

Cell therapy and gene therapy companies extended a trend from the second half of 2021 by making up 11 of the 21 financing deals in February. PharmaDJ expects this trend to continue throughout the year.

Investor preference for early-stage companies is apparent. In February, over three quarters of all financing consisted of seed (5%), angel (24%), Pre-A (19%) and Series A (29%) rounds.

TCR-T biotech the biggest fundraiser

TCRCure Biopharm received more than RMB300 million ($47.4 million) in series C financing. Established in 2016, the U.S. and China-based company has six technology platforms, including multiple immune cell engineering platforms, clinical immune monitoring platform, and an industry-scale vector production platform. The company is capable of developing various cell therapy products, such as CAR-T, TCR-T, CAR-NK, and TIL therapy.

TC-E202, TCRCure’s next generation TCR-T cell therapy in solid tumors, received IND approval from the CDE at the end of 2021. This is the first IND approval of TCR-T for cervical cancer in China and also the first PD-1 armed HPV TCR-T clinical trial IND approval in the world. TCRCure is also getting ready to submit its IND application of TC-E202 to the FDA.

TC-E202 is a cell therapy that can improve adoptive T cell therapy by incorporating immune checkpoint inhibitor.

It simultaneously encodes HPV16 E6 TCR and the variable region fragments of anti-PD-1. After viral transfections, T cells are able to express HPV16 E6 TCR and inhibit PD-1. When TC-E202 is injected into patients, TCR expressed on the surface of modified T cells can effectively recognize the tumor antigen HPV16 E6 of cervical cancer.

T cells activated by such tumor antigen are able to kill tumor cells. Meanwhile, the modified T cells can also secrete PD-1 single-chain antibodies, which can effectively eliminate the inhibition of tumor microenvironment. This increases the infiltration of T cells and enhances the efficacy of TC-E202 on solid tumors.

Only two other TCR-T therapies have received IND approval in China before TC-E202.

Xiangxue’s TAEST16001 received the first IND approval for a TCR-T among Chinese companies with a green light for the treatment of soft tissue sarcoma. The second to be approved is LionTCR’s LioCyx-M004, a genetically modified autologous T-cell therapy for the treatment of adults with hepatitis B virus-related hepatocellular carcinoma. The FDA has granted fast track designation to LioCyx-M004.

TCR-T has been making headway elsewhere. On Jan. 25, Immunocore announced the FDA approval of KIMMTRAK (tebentafusp-tebn) for the treatment of unresectable or metastatic uveal melanoma. KIMMTRAK is the first TCR-T therapy to receive regulatory approval and is also the first and only FDA approved therapy for the treatment of unresectable or metastatic uveal melanoma.

The approval of KIMMTRAK may stimulate further development of TCR-T therapies. At the same time, the FDA approval of Legend Biotech’s BCMA CAR-T is likely to boost the cell therapy industry in China.

Gene therapy company develops in vivo gene editing

YolTech Therapeutics deserves special attention among the gene therapy companies that raised funds in February. Domestic gene therapy companies often use virus vectors to deliver genetic material into cells to compensate for abnormal genes or produce a beneficial protein. But YolTech is focused on in vivo gene editing instead.

YolTech is primarily engaged in the development and optimization of CRISPR-Cas, base editing, and other gene-editing tools, as well as innovative improvements to mRNA production platforms and lipid nanocarrier (LNP) assembly processes. Its aim is to provide patients with in vivo gene-editing drugs for genetic and cardiovascular diseases.

Wu Xuanyu is the founder of YolTech and a well-known scientist in gene editing. During his time at Harvard Medical School, Wu found a new method based on CRISPR–Cas9 that can do highly efficient therapeutic gene editing of human hematopoietic stem cells. It is the first CRISPR–Cas9 gene therapy for hemoglobin diseases without virus vectors.

YolTech claims to be the first company in China to submit an IND application for an in vivo gene-editing product. So far, its products have been validated in varieties of animal models by doing gene editing 80% more efficiently in mouse liver models. Tests in non-human primates (NHP) have found that a single injection alone can knock down 60% to 70% of target genes in liver tissue. This efficiency rate is comparable to global leading companies.

In vivo gene editing is a cutting-edge technology but only a few sets of clinical data have been published about it.

On June 26, 2021, Intellia Therapeutics and Regeneron Pharmaceuticals announced positive interim data from an ongoing Phase 1 clinical study of their in vivo genome editing candidate NTLA-2001. The candidate is a single-dose treatment for transthyretin (ATTR) amyloidosis. It produced the first-ever clinical data supporting safety and efficacy of in vivo CRISPR genome editing in humans.

The data is derived from the first six ATTRv-PN patients of the phase I study. Single doses of either 0.1 mg/kg or 0.3 mg/kg of NTLA-2001 were administered systemically. Treatment with NTLA-2001 led to dose-dependent reductions in serum TTR, with mean reductions of 52% among the three patients in the 0.1 mg/kg dose group, and 87% among the three patients in the 0.3 mg/kg dose group, including one patient with a 96% reduction.

NTLA-2001 was found to be generally well-tolerated by the six patients included in the interim analysis, with no serious adverse events and no liver findings by day 28.

Many companies outside of China, such as Editas Medicine, Beam Therapeutics, Metagenomi, and Verve Therapeutics have joined in the race to develop in vivo gene editing products.

Big pharmas are also looking for opportunities to enter into this field.

At the start of the year, Pfizer and Beam Therapeutics inked an exclusive four-year research collaboration focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system.

Moderna and Metagenomi also established a collaboration to develop next-generation in vivo gene editing therapeutics shortly before Pfizer.

The financing of YolTech is likely to herald many more domestic gene therapy companies hopping on the gene editing bandwagon.

Keywords: Chinese investors cell and gene therapy in February
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