Logicbio Therapeutics Inc. hung out its shingle for public display, extending its fundraising with an oversubscribed $45 million series B led by publicly traded Arix Bioscience Ltd. and Orbimed with participation from Edmond de Rothschild Investment Partners, Pontifax and SBI Japan-Israel Innovation Fund. Formed in 2014, Logicbio last year closed a $5.4 million A round from Orbimed Israel.

In conjunction with the series B, the gene therapy specialist relocated from California to Cambridge, Mass., where it will continue to develop its genome-editing platform, known as Generide, and complete preclinical work to advance multiple programs targeting pediatric genetic diseases into the clinic.

The size of round was tailored to give Logicbio a runway of at least several years, deemed sufficient to advance two programs to IND filing and at least one of them through human proof-of-concept studies, according to Daniel O'Connell, Arix investment manager, who joined Logicbio's board.

Although, for the time being, the company is not disclosing lead indications, its technology and scientific publications point in the direction of liver diseases, which O'Connell described as "low-hanging fruit" in an effort expected to stretch across multiple organ systems.

"Logic's technology has several advantages which will allow it to be utilized in a multitude of different tissues," O'Connell told BioWorld. "What's nice here is that there's what we believe to be a tremendous amount of specificity that can be engineered into the technology. And using that, we're going to look beyond the liver, ultimately."

The differences in Logicbio's approach start with Generide, which achieves high levels of therapeutic proteins by harnessing homologous recombination to integrate therapeutic transgenes into a specific locus within the host genome. Generide uses a native promoter, without disrupting its normal function in the host genome, to enable permanent expression of a functional version of a missing or faulty gene – thus, offering a potential cure from a one-time therapy.

The second component of Logicbio's platform is a library of synthetic, non-pathogenic recombinant adeno-associated viral, or rAAV, vectors designed to provide a performance advantage over established clinical capsids.

During the past year, the company showed proof of concept in five animal models, completed prep work on the manufacturing side and generated data that position it to move into IND-enabling studies, according to Frederic Chereau, president and CEO.

'It's a very simple technology'

Gene therapy, immuno-oncology and anti-infectives represent three strategic areas where Arix has "gone hunting" for investment prospects, said CEO Joe Anderson. The Logicbio financing came together through a proactive process – "scoping the strengths and the weaknesses and where the opportunities lay" in gene therapy, he told BioWorld.

The directed effort led Arix to Logicbio, whose technology falls "somewhere between gene editing and gene therapy," Anderson added. The initial opportunity is focused on underserved pediatric populations, including infants – a group of patients that has been especially challenging for the field.

When traditional gene therapy techniques are applied in the youngest cohort of patients, the proliferation of cells in a child's growing tissue dilutes the therapeutic benefit. Yet, in rare genetic disorders such as inborn errors of metabolism, symptoms appear early and the disease progresses so rapidly that early intervention is needed to forestall major disability or death.

Because Logicbio's Generide technology hitches a therapeutic transgene to a native promoter without disrupting its normal function in the host genome, the desired outcome – permanent expression of a functional version of the missing or faulty gene – can be accomplished through a single treatment early in a child's life. The strategy also minimizes the risk of carcinogenicity associated with strong promoters used in traditional gene therapy approaches.

"It's a very simple technology," Chereau told BioWorld, suggesting that Logicbio arrived on the gene therapy scene at an ideal time, as the science was approaching the point of maturation, "where good companies will be able to deliver great drugs to patients in need."

Chereau, who spent a decade at Sanofi SA's Genzyme unit before leading regenerative medicine specialist Pervasis Therapeutics Inc. and rare disease firm Atyr Pharma Inc., said he was lured to Logicbio last year by the elegance of the scientific approach and the pedigree of the company's founders. Mark Kay, chief scientific advisor, is professor of pediatrics and professor of genetics at Stanford University School of Medicine. Co-founder Adi Barzel, who serves as Logicbio's acting chief science officer, is associate professor in the department of biochemistry at Tel Aviv University. Co-founder Leszek Lisowski, chief technology officer, is group leader at Children's Medical Research Institute in Australia. The trio collaborated to develop the company's two core technologies in the Kay Lab at Stanford, which licensed the technology to the startup.

In addition to Chereau, Kay and O'Connell, Logicbio's board includes Erez Chimovits of Orbimed Israel. In conjunction with the series B, they will be joined by Leon Chen, venture partner at Orbimed; Sofia Ioannidou, investment director at Edmond de Rothschild; and Tomer Kariv, founder and CEO of Pontifax.

For Arix, which gained a listing this year on the London Stock Exchange, where it trades as ARIX, Logicbio represents the ninth investment in little more than a year and the fourth in 2017. A day earlier, the company participated in the $20 million A round for Israeli startup Mitoconix Bio Ltd., and it expects to add another half-dozen holdings to its portfolio of life science companies "on the innovation end of the spectrum, where we see the real returns," Anderson said. (See BioWorld, Feb. 3, 2017, and June 28, 2017.)

These days, investors have an even greater impetus to fund potentially transformative science, given the drug pricing rancor that continues to dog the biopharma industry, he maintained.

"Logic fits absolutely squarely in the innovation category," Anderson said. "We've seen excitement around gene editing, and quite rightly so. These things come along very quickly, and you need to be alert to the changes when they do. We're seeing a sea change in the way regulation and manipulation of genes is being approached."

Established to resemble a venture firm but with a public structure, Arix has an extensive network of agreements with academic centers and accelerators that funnel opportunities – more than 700 in the past year, according to Anderson. The company has no intention of braking the process.

"We're absolutely thrilled that we're seeing such flow of high-quality opportunities," he said, suggesting the company's model offers access to early stage deals normally out of reach to public market investors. "It's great to see the plan unfolding."

"Genomics and gene therapies are themes of Arix, so Logicbio's name fits," Jefferies analyst Ken Rumph wrote in a flash note on Arix. "Since IPO [with some deals pre-dating], we estimate Arix is well advanced in deploying cash into business not otherwise publicly accessible."