$100 Million in Funds Will Help A Gene Therapy Startup Develop New Treatments

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According to a story from fiercebiotech.com, the gene therapy startup Generation Bio managed to fundraise $100 million in a little over a month. In its prior series A round of financing, the company raised $25 million. The company was started with the intent to develop new gene treatments that can be configured and delivered more like a typical drug, where the patient will take regular doses periodically. This innovation would be a significant improvement for patients and could help improve access to gene therapies.

The company plans to use the new funding to help push its first development candidates through studies that will grant them Investigational New Drug status. However, the company has not yet decided which candidates it will choose to develop first. Currently, Generation Bio is in the process of the developing gene therapies that use an alternative method of delivery called closed-ended DNA. Most currently available gene therapies use a virus to deliver the treatment, but the use of closed-ended DNA could be a safer alternative that would reduce the likelihood of immune system reactions.

Two likely treatments that will be selected for the funding are currently in the preclinical testing stage with mice and nonhuman primates. These experimental candidates are being developed to treat a variety of rare diseases, such as progressive familial intrahepatic cholestasisglycogen storage disease 1a, and phenylketonuria. The company is also in the process of fine-tuning the closed-ended DNA (ceDNA) delivery system for different parts of the body, such as the liver, lungs, central nervous system, and eyes.

The field of gene therapy is one that is constantly evolving, and drug companies are on the search for ways to improve gene therapies. Common issues include the fact that gene therapy’s effect is of relatively brief duration, requiring patients to receive multiple treatments. Generation Bio’s goal to develop therapies that can be delivered more like conventional drugs would help make this less of a problem.

There are also problems with the use of viral vectors, another issue that the company could help resolve. Viral vectors are not always precise with targeting, and they carry the risk of immune response. This can also cause a gene therapy to become less effective over time for a patient because the immune system learns to attack that is has encountered before, meaning that less of the viruses survive to deliver treatment. Hopefully, Generation Bio’s new treatments could be an answer to these challenges.


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