Elevatebio Inc. CEO David Hallal told BioWorld that the five-member syndicate of investors behind his firm's whopper $150 million series A round "all have been on the other side of the table" in cell and gene therapy, and thus understand the potential value of building a portfolio of new companies in both areas by way of partnerships with academic researchers, medical centers and entrepreneurs.

Cambridge, Mass.-based Elevatebio's business model centers on an effort called Basecamp that will put together single- and multiproduct enterprises in cell and gene therapies by putting in the hands of the founders centralized, bench-to-bedside capabilities that include scientists, manufacturing facilities (in a space that measures more than 100,000 square feet), and drug development as well as commercialization expertise. "We know one of the most significant barriers [in cell and gene therapies] is access to high-quality process development and manufacturing," said Hallal, formerly CEO of New Haven, Conn.-based Alexion Pharmaceuticals Inc. "Demand is far outstripping capacity. It will through the end of this decade and it will through the end of the next."

Areas of focus for Elevatebio include immunotherapy, regenerative medicine and viral-based in vivo gene therapy. The first portfolio entrants will be disclosed in "the next few weeks," Hallal said. "We believe in being agnostic from the perspective of transformation," he added. Although immuno-oncology makes for a prime area of interest, "we also want to be thoughtful around other disorders where we see something that we believe can change the world and transform the lives of patients," which means not only cancer but also "an entire set of devastating and life-threatening disorders." Research will be chosen "across a spectrum of stage, from late preclinical to even advanced stages of development," with particular scrutiny given to feasibility. Investors often "come across science that is jaw dropping but there may be execution challenges with how long and how much money it may take to ever get to a patient," he noted.

Carefully stratifying portfolio firms to represent early and late-stage efforts is "the ideal approach, because we feel like that really diversifies your portfolio and enables your R&D and manufacturing team to be applied to every stage of development, and yet not be overly exposed to any single stage," Hallal said. "We're in the earliest days of the cell and gene therapy revolution," as researchers realize that "so much more can be done faster" with the kind of access that Basecamp aims to provide, he said.

The fields of cell and gene therapy have exploded thanks to "the progress of pioneers and innovators in the space," among them Elevatebio's chief scientific officer (CSO), Mitchell Finer, who has more than 30 years of experience in cell and gene therapy, including five years as the first CSO of Bluebird Bio Inc., of Cambridge, Mass. He was involved in the first CAR T therapies in the 1990s. Elevatebio, Hallal said, has an edge through its "unique access to talent. It's the people, it's the team," with the likes of Finer attracting the best and brightest.

Cell therapies of various kinds continue to draw Wall Street's attention. Canaccord Genuity analyst John Newman reported leaving the Allogeneic Cell Therapy Summit earlier this month with "incremental learnings about the different cell types. For example, CRISPR/Cas9 can be potentially used to create multiplex edited CAR T cells with a lesser degree of gene translocation, and the fast/cheap platform allows for rapid development of products from conceptualization to mice models. Gamma delta T cells are less potent killers than alpha beta T cells, and companies are either taking advantage of their natural strengths to eliminate minimal residue disease or modifying them to improve tumor killing. Natural killer [NK] cell characteristics are dependent on the source, and monoclonal antibody combination therapy seems attractive." What's more, NK T cells "have shown early encouraging autologous data in neuroblastoma patients, with allogeneic studies starting later this year" and companies with efforts in the space will deliver data this year and next, he noted.

MPM companies on the move

Wainwright analyst Joseph Pantginis recently singled out cell therapy player Biotime Inc., of Alameda, Calif., which is expected to launch a rebranding of the company "with the new laser-like focus on becoming a true leader in cell therapy, including a potential name change of the company." Biotime in March closed the merger with Fremont, Calif.-based Asterias Biotherapeutics Inc. "All focus is now on driving the cell therapy pipeline forward with three clinical-stage therapies underway, led by Opregen," a retinal pigment epithelium cell transplant therapy in development for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA), Pantginis wrote in a report last week, adding that he expects updates on the compound in October at the American Academy of Ophthalmology meeting in San Francisco. Earlier this month at the Association for Research in Vision and Ophthalmology annual meeting in Vancouver, British Columbia, Biotime unveiled results from the phase I/IIa study showing that treatment with Opregen continued to be well-tolerated and in some patients, clinicians found signs of structural improvement in the treated areas of the retina. Data from fourth-cohort patients with earlier-stage dry AMD and smaller areas of GA remained encouraging, with indications of the continued presence of the transplanted Opregen cells and improvements in visual acuity, Biotime said. "As for the OPC-1 clinical trial, we are looking forward to the next steps of clinical development following upcoming FDA discussions and potential improvements of the manufacturing process," he said. OPC-1 is an oligodendrocyte progenitor cell therapy in a phase I/IIa multicenter study for acute spinal cord injuries that has been partially funded by the California Institute for Regenerative Medicine.

In gene therapy, Wainwright's Debjit Chattopadhyay tipped his hat to Amsterdam-based Uniqure NV, with AMT-061 in hemophilia B, hailing the safety profile, established manufacturing and compelling clinical outcomes. "AMT-061 is positioned to be the first and best-in-class program in hemophilia B, which could compel Pfizer Inc. to have second thoughts in its pursuit of a me-too program," given that the pharma giant is handicapped by such factors as intellectual property and T-cell activation-related reproducibility. After seeing data at the North American Society on Thrombosis meeting in New Orleans this month, "we consider AMT-061 to be one of the more de-risked gene therapy programs with a clear path to a dominant commercial position." AMT-061 consists of an AAV5 viral vector carrying a gene cassette with the Padua variant of factor IX. The company is enrolling severe and moderately severe hemophilia B patients in the phase III pivotal study called Hope-B. In February 2019, Uniqure said it had treated the first patient in the trial and plans to complete patient enrollment before the end of this year.

MPM-backed companies have turned up in the news plenty lately. Last month, Cambridge, Mass.-based Dyne Therapeutics Inc., founded and seeded last year by Atlas Venture, completed a $50 million series A with Forbion and MPM. Dyne is working on therapies for patients suffering from myotonic dystrophy type 1, a rare and inherited disorder with life-limiting complications that affects about 40,000 people in the U.S. (See BioWorld, April 4, 2019.)

In March, Cambridge, Mass.-based startup Trinetx Inc. pulled down $40 million in a series D to better match patients with clinical trials and real-world evidence efforts. Merck Global Health Innovation Fund, the $500 million digital health-focused strategic fund of Kenilworth, N.J.-based Merck & Co. Inc., led the financing, which boosted the total raised by the company to more than $102 million. (See BioWorld, March 15, 2019.)

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