Neurogene nets $68.5M to advance gene therapies for rare neurological diseases

Though the company is based in New York, about one-third of its employees are in Houston, where it plans to build its manufacturing facility. (Pixabay/mpewny)

Neurogene raised $68.5 million in its series A round to push forward multiple gene therapy programs, invest in new technology and build its own viral vector manufacturing site for its therapies. The plan is to move its lead programs, for Aspartylglucosaminuria (AGU) and a rare form of Charcot-Marie Tooth disease, into IND-enabling studies this year, with clinical trials slated for 2020. 

It draws its funding from Samsara BioCapital, EcorR1 Capital, Cormorant Asset Management, Redmile Group and an undisclosed leading healthcare investment fund, according to a statement.

Neurogene is working on treatments for AGU and CMT4J, a relatively rare form of Charcot-Marie Tooth, because these are pretty horrible lysosomal storage diseases,” said Neurogene CEO Rachel McMinn. “They have a very rapid onset for kids who develop it early on.” 


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AGU is caused by a mutation in the AGA gene, which codes for an enzyme of the same name: aspartylglucosaminidase (AGA). Children with AGU produce no AGA, or very little of it, which results in “a whole bunch of storage material building up across the body in many tissues,” McMinn said. “The biggest issues are within the CNS and people all present with developmental delay, which can lead ultimately to a shortened life span.” 

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As for CMT4J, CMT is an umbrella term for a group of disorders in which the motor and/or sensory peripheral nerves are affected. Some CMTs are debilitating, but people can live quite a long time, McMinn said. But CMT4J can be “quite progressive,” lead to respiratory distress and ultimately, prove fatal, she said. 

In addition to its AGU and CMT4J programs, Neurogene will also advance two programs for undisclosed lysosomal storage diseases.

The company is also investing in “novel technologies for indications not addressed by traditional gene therapy approaches,” because there are limitations to the reach of gene therapy. Though gene therapy is an “amazing tool,” McMinn said it is still “a little bit crude.” Some genes—such as the dystrophin gene, which malfunctions in boys with Duchenne muscular dystrophy—are too large to fit inside viral vectors, while other genes can be toxic if delivered in too high a dose. McMinn did not disclose just what, exactly, Neurogene is working on, but said that the company is investing in technologies it thinks will complement gene therapy. 

Neurogene has been hiring “pretty steadily” and will soon hit 20 staffers. Though the company is based in New York, about one-third of its employees are in Houston, where it plans to build its manufacturing facility. As Neurogene forges ahead, it will add to its manufacturing team as well as to its medical and clinical teams.

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