gene therapy companies

Gene Therapy Companies On The Rise

gene therapy companies

Passage Bio Files for IPO to Advance Gene Therapy

Passage Bio is one of the gene therapy companies headquartered in Philadelphia that develops treatment for rare monogenic CNS diseases. They are planning to raise $125 million through an initial public offering, according to co-founder and pioneer researcher James M. Wilson, MD, Ph.D. They intend to use the net proceeds to advance their lead products in clinical trials, discovery and candidate selection stage programs, and general corporate purposes.  

Passage Bio was formally launched with $115.5 million in Series A financing in February 2019. They plan to develop five therapeutic candidates incorporating adeno-associated virus (AAV) technologies which were developed by Wilson and his team at the University of Pennsylvania (Penn) and its Gene Therapy Program (GTP). Three of these are in IND-enabling phases and are slated to begin clinical development. They also plan to launch a Phase I/II trial for PBGM01 by mid-2020, making clinical data available in the first half of 2021. In addition, they will launch two other programs in the first half of 2021PBFT02 which is designed to target the gene encoding the lysosomal protein progranulin (PGRN) for the treatment of frontotemporal dementia and PBKR03 which will target the gene encoding the hydrolytic enzyme galactosylceramidase (GALC) for the treatment of Krabbe disease. Clinical data for both candidates is expected in the second half of 2021.

Passage Bio’s authorized capital stock will consist of 300 million shares of common stock and 10 million shares of undesignated preferred stock upon the completion of the IPO.

Beam Therapeutics Raises $180M in IPO

American chemist and biologist David R. Liu, Ph.D., Chinese-American biochemist Feng Zhang, Ph.D., and American pathologist and molecular biologist Keith Joung, MD, Ph.D. are the co-founders of Beam Therapeutics and the base editing drug developing firm was able to raise $180M in IPO. Base editing originated with Alexis Komor, Ph.D., and Nicole M. Gaudelli, PhD., two postdocs in Liu’s laboratory.  They were able to devise a pair of molecular machines that can correct the most known disease-related mutations in the human genome. With this technology, Liu was prompted to establish a new company along with Zhang and Joung. 

The name Beam was suggested by a pediatric oncologist at Stanford University School of Medicine Agnieszka Czechowicz MD, Ph.D., and evoked from a laser, precision technology. It also stands for Base Editing and More. Their novel approach to base editing, particularly single-letter genome editing, brings a wide range of potential for the treatment of diseases making them one of the gene therapy companies to watch out for.

Beam Therapeutics has grown to more than 100 employees since it was founded in 2017 covering elements of base editing, and additional gene editing technologies and delivery modalities. They have raised approximately $224 million in capital from various fundings. Their goal is to discover, develop, manufacture, and commercialize a new class of medicine utilizing base editing technology to treat patients suffering from serious diseases.

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