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Launch of Rectify Pharmaceuticals

Country

United States

A new biotech company with plans to target ABC transporters, one of the oldest families of proteins, has been launched with $100 million in Series A financing by US-based Atlas Venture and three other groups. Rectify Pharmaceuticals Inc will develop small molecule drugs aimed at restoring dysfunctional ABC transporters to a normal state. These transporters play an essential role in ferrying organic and inorganic molecules across cellular membranes.

Genome editing to be investigated for ocular diseases

Country

United States

A new collaboration between Intellia Therapeutics Inc and the French ophthalmology company SparingVision SAS will seek to apply genome editing technology to the treatment of diseases of the eye. Announced on 13 October, the partnership will use Intellia’s in vivo Crispr/Cas9-based platform to investigate up to three ocular targets.

Exosomes delivered proteins to the brain in animal

Country

United Kingdom

ReNeuron Group Plc has reported that exosomes were able to deliver a therapeutic protein to the brain of an animal and function better than the supply of the protein alone. Exosomes are intracellular vesicles that carry a variety of proteins and genetic material between cells. ReNeuron is exploring their use as possible delivery vehicles for therapies targeting neurological diseases.