Case Study

Spark Therapeutics Taps Genesis Engineers For New Center Of Operations

Source: BioProcess Online
Spark Therapeutics Taps Genesis Engineers For New Center Of Operations

By Genesis Engineers and Spark Therapeutics

Size: 20,000 sq. ft.

Services: Process Design and Scale-up, Engineering, Construction, Commissioning

Situation                  

Spark Therapeutics (“Spark”) had identified a location for its new center of operations, which required laboratory and early-stage commercialization build-out. Spark’s timeline for occupancy was nine months and required an experienced partner to assist with the project based on their existing research-based facilities at Children’s Hospital of Philadelphia (“CHOP”). Recognizing that the nine-month timeline was extremely tight, Spark turned to Genesis Engineers (“Genesis”), which had done exceptional work for CHOP previously and thus recognized Genesis’ demonstrated process experience with biotech GMP manufacturing & scale-up.

Background                       

Spark’s gene therapy technology originated in the research laboratories of renowned CHOP, and when CHOP decided to make a direct investment in launching Spark Therapeutics, Spark needed to move into an independent facility.

The CEO and team believed in the synergy of staying in the University City section of Philadelphia and chose a site on the 13th floor of a new building being constructed at 3737 Market Street, in a neighborhood known as the University Science Center. Genesis Engineers had worked on a recent project at CHOP and was recommended to the Spark team.

The company has several gene therapy treatments in the pipeline, at all stages of pre-clinical and clinical development. Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing one-time, life-altering treatments for debilitating genetic diseases.

Solution

Process Design And Timeline

Every project has a driver. This one had several, including a less than one year timeline from start to opening the doors, but you have to choose one. For Spark, the process operation piece was the driver.”

-Mike Dapkunas, Project Principal, Genesis

Genesis Engineers has a wide range of capabilities that make the company a unique engineering and construction management partner for early-stage biotechs. The firm’s process design experience is excellent. Thirty-year biotechnology veteran Norm Goldschmidt’s guidance was required to design a scale-up manufacturing suite capable of early-stage commercialization for their current clinical product development as well as provide capabilities for the future products in their pre-clinical and clinical pipeline. Spark required R&D, Process Development and QC laboratories, plus manufacturing space.It was imperative that Spark’s partner have strong cleanroom and process design experience coupled with a deep understanding of the requirements for regulatory and licensing.

In addition, the Genesis Project Architect, Mike Marone, had a leadership mentality and a triple aptitude for design capability, laboratory expertise, and process understanding — a rare combination. The benefit to Spark was the ease of communication; Geneisi Engineers had one individual with many of the answers; there was no need to consult three different sets of expertise.

With Norm Goldschmidt and Mike Marone collaborating with the Spark Core Team, decisions were focused on uni-directional flow for materials and personnel flows, disposable technology, and isolation suites configured for flexibility.

One of the earliest decisions that had to be made was whether to build out the new facility using a modular approach or to design a traditional stick-built facility. Genesis made the decision making around this question quite visual by bringing in the vendors for in-depth presentations with the Spark teams, some of whom were unfamiliar with modular materials and construction. It was soon clear that the aggressive schedule mandated modular construction. (The CEO and CHOP wanted the new doors open on October 7, 2014.) AES was chosen for this portion of the project.

One of the earliest challenges (January 2014) in design was how to account for accommodations for unexpected growth spikes in the Spark pipeline of therapies, which, depending on R&D results, could expand in any direction, affecting headcount and workflow in the new space. Genesis dealt with this by building in extra R&D and laboratory capacity within the confines of the core shell and 13th floor footprint of a high- rise building in Center City, Philadelphia.

In January 2014, a primary goal was tackling how to lead this group of scientists who only had experience in a 20-year-old retrofitted lab setting at CHOP. How could they help this team to visualize the sleek, metal-based (versus wooden casework) look of today’s biotech companies? Initially there were six people on the Spark team, but Spark was now building a facility for a planned headcount of 60+ employees. Drawing information out of core team leads — clinical leads, administration leads, and quality leads — was critical, and Genesis quickly developed a culture of trust needed to configure those requirements into a floor plan that would satisfy three vastly different mentalities: Manufacturing, Laboratory, and Administrative teams.

To effectively lead Spark though a visualized process design scale-up, Genesis drew upon their earlier experiences working with early stage companies and knew how to let the client take part in decisions about everything that was accessible and understandable : layout, color, people flow, and laboratory requirements. This approach increased Spark’s comfort level when Genesis made process scale-up decisions that were more difficult to visualize.

Floor plans and bubble diagrams became the main visual form of communication during the weekly meetings held by the Genesis-Spark teams, right from the earliest days of January 2014. The Spark team wanted to became involved in the details they could see and control, and Genesis welcomed the side-by-side decision process which even included a field trip to NYC’s furniture district to choose office furniture.  

The top-line results of Spark’s Phase 3 trial for a rare form of inherited blindness are expected in October 2015, and if data are positive, Spark hopes to become the first company to receive FDA approval for a gene therapy treatment for a genetic disease.

Plans are underway for a much-needed expansion of office space, as the company has grown from the planned 62 to more than 80 employees. Genesis and Spark are working together on this expansion project.

BACKGROUND

Genesis Engineers (www.geieng.com)

Founded by Bernie Friel, today’s Genesis Engineers began as Genesis Engineering & Design, a mechanical design firm servicing the pharmaceutical industry. In 1997, a merger with Schertzer Engineering Associates, an electrical design firm, led the way to Genesis/SEA, a full service MEP engineering and design firm. Then, in 2001, the addition of three new partners, each with over 20 years of experience in the areas of project management, construction management, and financial management, the company transformed into Genesis Engineers, a full service project delivery firm.

In 2013, the addition of a new partner and the growth of an architectural staff, in-house architectural services are now being provided. Today, Genesis’ engineering-led focus and a staff of over 100 talented individuals has built an excellent reputation for leading the delivery of technical based projects.

Spark Therapeutics (www.sparktx.com)

Spark Therapeutics is developing one-time, life-altering gene therapy treatments to transform the lives of patients and re-imagine the treatment of debilitating diseases.

Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing one-time, life-altering treatments for debilitating genetic diseases. They are initially are applying their integrated platform, which was developed over two decades at The Children’s Hospital of Philadelphia (CHOP), to treat rare diseases where no, or only palliative, therapies exist.

The gene therapy vectors used in their programs are engineered using an adeno-associated virus, or AAV, which are designed to encapsulate a genetic sequence and have been demonstrated to be effective vehicles for delivering genetic material into targeted cells, providing unique advantages over alternative therapeutic approaches. Their most advanced product candidate, SPK-RPE65, is intended to treat rare blinding conditions caused by mutations in the RPE65 gene, and, in earlier clinical studies, showed promising evidence of its ability to safely and durably restore vision in patients. In November 2014, they announced that SPK-RPE65 received breakthrough therapy designation from the U.S. Food and Drug Administration. Breakthrough therapy designation is intended to expedite the development and review of investigational therapies for serious or life-threatening conditions where initial clinical evidence demonstrates a significant improvement over existing therapies. 

Spark is currently conducting a fully-enrolled, Phase 3 pivotal clinical trial of SPK-RPE65 and anticipate reporting final results from this trial during October 2015. They are leveraging SPK-RPE65 to address a broad spectrum of other inherited retinal dystrophies, or IRDs, caused by different genetic mutations. Our first such follow-on product candidate is SPK-CHM for the treatment of Choroideremia, for which Spark has initiated a dose-escalating, Phase 1/2 clinical trial. Spark has also established a pipeline of gene therapy candidates to treat hematologic disorders and neurodegenerative disorders, including SPK-FIX, a program for the potential treatment of hemophilia B through a global collaboration with Pfizer.

Spark began operations in October 2013  when they acquired and in-licensed intellectual property and the development and commercial rights to clinical and preclinical programs advanced at CHOP. Many experts from the gene therapy center at CHOP have since joined the Spark team, including the center’s founding director Dr. Katherine A. High.

Since their launch, Spark has secured over $280 million in financings, including an initial public offering in January 2015 and corporate collaborations with both Pfizer and Genable Technologies, providing the capabilities and resources needed to potentially deliver innovative medicines for those in need.

Spark is proud of its roots in Philadelphia and currently occupies its 28,000 square foot office, lab, and manufacturing facility at the University City Science Center in West Philadelphia. As our proprietary manufacturing process is critical to support potential commercial demand for multiple gene therapy product candidates — including our lead Phase 3 program for SPK-RPE65 – this facility includes our own state-of-the-art current Good Manufacturing Practice (cGMP) facility to manufacture clinical and commercial grade AAV vectors.