REGENXBIO's Gene Therapy RGX-121 Demonstrates Strong Clinical Performance in MPS II and Receives FDA RMAT Designation

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Recently, REGENXBIO announced that the investigational gene therapy RGX-121 met the primary endpoint in a pivotal clinical trial for the treatment of Mucopolysaccharidosis II (MPS II). The levels of D2S6 in patients' cerebrospinal fluid decreased by an average of 86%, approaching normal levels. D2S6 is one of the key biomarkers of brain disease activity. Previously, positive data were reported from the Phase I/II/III trials of RGX-121 for MPS II patients under the age of 19, and there have been reports showing that RGX-121 has provided sustained benefits to patients for up to three years. These results support the company’s plan to submit a Biologics License Application (BLA) to the U.S. FDA via the accelerated approval pathway in 2024. Notably, RGX-121 has received Orphan Drug Designation, Rare Pediatric Disease Designation, Fast Track Designation, and Regenerative Medicine Advanced Therapy (RMAT) Designation from the U.S. FDA.

Among them, RGX-121 is a gene therapy designed to use the AAV9 vector to deliver the transgene encoding human iduronate-2-sulfatase (IDS) to the central nervous system (CNS). Delivering the IDS transgene into CNS cells can provide patients with a permanent source of IDS secretion, which is capable of crossing the blood-brain barrier, thereby correcting or repairing genetic defects or functional abnormalities in surrounding cells.

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Recently, Metagenomi, an emerging gene-editing therapy development company, stated in its filing with regulatory authorities that it plans to raise nearly $87 million through an IPO. Unlike other biotech companies that have completed IPOs this year, Metagenomi is a platform-based company without any clinical-stage development projects. Its goal is to develop a differentiated next-generation toolbox for gene-editing systems.

It was reported that Metagenomi had signed several high-profile deals. In 2021, the company entered into a strategic R&D collaboration with Moderna, which would combine Metagenomi's novel CRISPR gene editing tools and leverage Moderna’s advanced mRNA platform and lipid nanoparticle (LNP) delivery technology to develop curative therapies for patients with serious genetic disorders. In 2022, the company completed a $175 million Series B financing. Shortly after, in November 2022, IONIS also reached a collaboration with the company, which could be worth up to $300 million.

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Recently, Chengdu Maikang Biotechnology Co., Ltd. announced the completion of a nearly 300 million yuan C-round financing. This round of financing was exclusively invested by Shenzhen Capital Group's Manufacturing Transformation and Upgrading New Materials Fund (referred to as "Shenzhen Capital Group").

Among them, the recombinant shingles vaccine (CHO cells) independently developed by Maikang Biotech adopts a domestically innovative technical route in China and overcomes the "new adjuvant" bottleneck technology. This pipeline is about to enter Phase III clinical trials, which can promote the body to produce high-level humoral immunity and cellular immunity against the varicella-zoster virus, preventing the onset of shingles, with broad market prospects. Similarly, the重磅 candidate product, recombinant respiratory syncytial virus vaccine (CHO cells), which is based on urgent clinical needs, has submitted a pre-IND, and its R&D progress is leading in China.

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Recently, Krystal Biotech announced that a case of Vyjuvek treating ocular diseases in DEB patients was published in the prestigious medical journal, *The New England Journal of Medicine* (NEJM). This marks the first instance of using this gene therapy to treat ocular complications in DEB patients, representing an important milestone in expanding the use of Vyjuvek.

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On February 8, 2024, Ionis Pharmaceuticals announced that the FDA had granted Fast Track designation to Eplontersen, its investigational therapy for treating adult transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), which is being developed in collaboration with AstraZeneca (AZ).

Among them, Eplontersen is an investigational antisense oligonucleotide drug administered once a month via subcutaneous injection by the patient themselves. It utilizes ligand-conjugated antisense oligonucleotide (LICA) technology, which conjugates ASO drugs with ligand molecules capable of binding to specific receptors on the cell surface. LICA technology can effectively deliver ASOs selectively to cells expressing these receptors. Eplontersen can be used to inhibit the production of TTR protein, thereby treating hereditary and non-hereditary amyloidosis polyneuropathy. This drug has been granted orphan drug designation by the U.S. FDA.

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