Gene Therapy Biotech Secures $37.5 Million in Venture Funds

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Applied Genetic Technologies Corp., a biotechnology company in Gainesville, Florida, gained $37.5 million in series B funds, the second round of financing after initial start-up. Alta Partners and S.R. One Ltd led the financing, with new investor Osage University Partners joining existing investors InterWest, Intersouth Partners, and MedImmune Ventures in the round.

Applied Genetic Technologies develops gene therapies using a harmless, engineered adeno-associated virus to deliver replacements for malfunctiong genes causing diseases. The company’s process removes viral gene from the adeno-associated virus to deliver the therapeutic gene. The replacement gene can act as a regulator of protein expression or promote expression of beneficial proteins in human tissue.

The proceeds of the round are expected to support Applied Genetic Technologies’ phase 2 clinical trial of a gene therapy to treat alpha-1 antitrypsin deficiency, a condition where the body does not make enough of a protein that protects the lungs and liver from damage. The condition can lead to emphysema and liver disease. Current treatments that involve infusions of alpha-1 antitrypsin protein purified from plasma, says the company, are expensive and limited by the low number of human plasma donors.

The financing is also expected to fund gene therapies for two rare retinal diseases, achromatopsia and x-linked retinoschisis. Both of these programs are in preclinical stages.

Achromatopsia is an inherited condition affecting about 1 in 33,000 Americans. People with achromatopsia experience color blindness, visual acuity loss, extreme light sensitivity, and involuntary eye movements.

X-linked retinoschisis is a juvenile genetic eye disorder that affects the retina mainly of boys, but can continue into adulthood. Damage to nerve tissue in the retina impairs visual acuity.

Read more:

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• Leukemia Genetic Signature, Drug Candidates Identified

Hat tip: Fortune/Term Sheet

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