La Jolla Acquires Rights To Next-Generation Gentamicin Derivatives To Advance Therapies For Duchenne’s

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by Isaura Santos |

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La Jolla Pharmaceutical Company, a drug development company working on therapies that address life-threatening diseases such as Duchenne muscular dystrophy, recently announced it has entered into an exclusive agreement to acquire the Indiana University Research and Technology Center’s (IURTC) intellectual property rights that cover the derivatives of their next-generation experimental therapy gentamicin.

Gentamicin is currently one of the most frequently prescribed hospital antibiotics, despite its tendency to cause toxicity in the kidneys. The IURTC’s technology includes the utilization of gentamicin derivatives as antimicrobial agents and with the potential of significantly reduced toxicity.

La Jolla enrolled into a second agreement with IURTC and the University of Alabama at Birmingham (UAB) to use these next-generation compounds in order to address treatment for rare genetic diseases like Duchenne muscular dystrophy and cystic fibrosis. Gentamicin might correct some human genetic mutations that cause rare genetic disorders through inducing lack of fidelity in the process of gene transcription.

However, even with such favorable efficacy characteristics, the advancement of gentamicin as a treatment for genetic diseases has been limited because of its toxicity profile.

La Jolla initially selected two candidates, LJPC-30Sa and LJPC-30Sb; both of which are purified components of the commercialized gentamicin product. LJPC-30Sa and LJPC-30Sb have gentamicin’s same biologic activity, but apparently lack the usual kidney toxicity associated with it. The company aims to advance a development strategy to address serious bacterial infections and rare genetic disorders caused by stop codon mutations like cystic fibrosis and Duchenne muscular dystrophy. La Jolla will proceed with a Phase 1 clinical trial after submitting an Investigational New Drug application (IND).

“We are very pleased to gain access to this intellectual property covering next-generation gentamicin derivatives. The use of aminoglycoside antibiotics has been limited primarily by treatment-related toxicity. We believe that our next-generation gentamicin derivatives may retain the activity of gentamicin, but improve the therapeutic window, thereby improving the outcome for patients requiring antimicrobial agents and potentially creating new opportunities for the treatment of rare genetic disorders,” said George F. Tidmarsh, CEO and President of La Jolla.

Duchenne muscular dystrophy is an inherited disorder caused by a defective gene called dystrophin. The disorder is characterized by a progressive skeletal muscle weakness that leads to chronic inflammation and the loss of muscle cells and tissue, compromising locomotion. Duchenne muscular dystrophy has a rapid progression and affects mainly boys.

Read More Recent News About Advancements In Research To Address Duchenne Muscular Dystrophy

Researchers at the University of California San Diego recently published in the journalBMC Research Notes an approach to assess possible dysregulated mechanisms linked to Duchenne muscular dystrophy. The study is entitled “Dysregulated mechanisms underlying Duchenne muscular dystrophy from co-expression network preservation analysis.