The growth of cell and gene therapies

High-value cell therapy deals are driving investment in the cell and gene therapy field, but new approaches such as gene editing are also beginning to contribute.

NurExone has created ExoTherapy, a cutting-edge exosome-based drug-delivery platform, and is currently developing its lead product, ExoPTEN, as a novel therapy for acute spinal cord injuries.

By engineering naturally existing arrestin domain-containing protein 1 (ARRDC1)-mediated microvesicles (known as ARMMs), Vesigen Therapeutics’ platform enables the effective delivery of novel biotherapeutics to specific tissues and cell types.

As Pandorum Technologies’ bioengineered ‘liquid cornea’, Kuragenx, is about to enter a first-in-human study for vision restoration in patients suffering from corneal blindness, the company is extending its proprietary technology platform to develop advanced exosome-based therapeutics for lung and liver regeneration.

bit.bio is a synthetic biology company that uses its cell identity coding platform to reprogram induced pluripotent stem cells to create any human cell type.

Pennsylvania-based Ocugen is a fully integrated, patient-centric biotech company focused on the development of vaccines in support of public health, and gene and cell therapies targeting unmet medical needs, all based on its three scientific platforms.

UK-based Broken String Biosciences is developing a commercial analytics platform for CRISPR therapeutics safety, a space in growing need of a gold standard solution for measuring off-target gene editing.

With its unique and proprietary genetically modified adipocyte (GMAC) platform, CellGenTech is developing novel ex vivo gene therapies and has achieved the first-in-human study of an adipocyte-based gene therapy.

The biotech firm’s polyclonal anti-cancer therapy is based on an ex vivo approach and avoids the use of toxic immune-depleting chemotherapy.