The Role of Incubators in Cell and Gene Therapy Development

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Working with incubators is a good idea for any biopharmaceutical company that is looking to expand its research, service, and consulting capabilities. Using incubators can be particularly helpful for companies that are developing cell and gene therapies (CGTs). Audrey Greenberg, co-founder and executive managing director of The Discovery Labs, and Christopher Potts, senior IP counsel and business partner at Lonza Pharma & Biotech, share some insights into incubators and the role they play in CGT development with BioPharm International.

Benefits

BioPharm: What are the benefits of incubators focused on CGT development?

Greenberg (Discovery Labs): Incubators allow for efficient use of capital for cash-crunched startups, allowing them to share equipment, resources, and space, so that they can spend their capital on innovation rather than administration.  Incubators also offer other valuable resources that can expedite the commercialization process. Some even offer capital investment.

For example, the Discovery Labs incubator, Unite IQ, provides access to manufacturing capacity, different technology platforms, raw materials, drug substance and drug product, plasmids, viral vectors, process development, as well as CRO [contract resource organization] services, clinical trial capabilities, and IND [investigational new drug] writing/submission services. In addition, it can provide access to legal counsel, grant-writing expertise, and business formation consulting so that innovators can turn scientific discoveries into profitable businesses and grow their research capabilities. 

Unite IQ can allow companies to grow from bench- to commercial-scale on the same campus.

Potts (Lonza): A key benefit is that incubators (in all industries but particularly for CGTs) allow members to leverage resources that can help emerging companies fill expertise and resource gaps. Top of mind is in the area of laboratory space equipment for early process development and proof of concept work. But the value add for some incubators extends to offering subject matter expertise in peripheral issues to emerging cell and gene therapy businesses. For example, they can provide subject matter experts in payer/reimbursement models to allow incubator members to develop robust business models that navigate the complex global payer systems and reimbursement strategies. As CGT incubators develop, they should seek to provide access to subject matter experts in a range of business support functions so that emerging companies can focus on the core of their technologies and business models, and not have to burn resources on retaining SMEs [subject matter experts]to round out their development.

Another major advantage of incubators in cell and gene therapies is to provide access to lab space, equipment, and manufacturing capabilities.

BioPharm: Why should companies invest in a CGT incubator?

Potts (Lonza): When you look at the top incubators in our field, you see a direct alignment between early stage researchers, funding, and contributing companies. This works both ways to provide researchers avenues in which to develop new businesses, with direct access to larger institutions that may provide expertise and funding, but also provides the large institutions with a first look at promising early stage technology and businesses. This alignment can speed up the early funding process, which in turn, speeds the time to clinic. Additionally, in many large companies, R&D groups have a gap in providing early stage foundational research that could form the basis of a new therapeutic or enabling technology. Large companies can fill this gap by enabling the incubation of small entities to bring foundational ideas toward commercialization.

Greenberg (Discovery Labs): Large companies who are not entrepreneurial due to their size or culture, can partner with or invest in an incubator that offers a front-row seat for innovation and investment opportunities. Investing in companies that are members of an incubator is based on that company’s specific technology, management team, and execution plan.

Successes

BioPharm: Do you have any success stories regarding how incubators/collaboration have sped the development of new therapies from lab to clinic?

Greenberg (Discovery Labs): The cohabitation and collaborative aspect of the Unite IQ incubator allows for better problem solving and resources to quickly address issues as they arise.  Incubators help bridge what some have called the ‘valley of death,’ or the gap between idea and commercialization. Academic scientists, who may lack deep business knowledge, often struggle to transform research and ideas into commercially viable therapies.

Many incubators have sprung up to help bring therapies from bench to bedside including Pfizer’s Centers for Therapeutic Innovation, Johnson & Johnson’s JLABS, and Bayer’s CoLaborator, as well as many private and public spaces around the US, particularly in biopharma hotbeds like Philadelphia, Boston, and San Francisco. Other examples include Pennovation in Philadelphia and Harvard Life Labs in Cambridge.

Numerous studies attest to the positive impact that incubators have on local economies, based on the survival rate of the companies and the number of jobs created from innovation and successful technology transfer. The environment of incubators fosters an entrepreneurial climate which leads to greater success. An example would be Pennsylvania Biotechnology Center, a nonprofit life sciences incubator-accelerator in Bucks County, PA that is partnering with The Discovery Labs in King of Prussia, PA Biotech’s site offers laboratory and office space to 38 start-up biotech companies. Some of these companies have seen their drug discoveries and medical devices approved by FDA, have reached over $2 billion in combined valuations, and have been acquired by major pharmaceutical companies. The Pennsylvania Biotechnology Center (PABC) has consistently outperformed peers in markers of success, including the number of successful innovators that have graduated from the program. In 2018, PABC was named one of the most successful biotechnology incubators in the country, according to an independent study by the International Business Innovation Association (1).

Three FDA-approved medical products or treatments have been developed by companies at PABC. In addition, eight individuals working at PABC have discovered drugs that have been approved by FDA, including Mike Sofia, PhD, who won the 2016 Lasker Prize for leading the discovery of a cure for hepatitis C (2); and Kunwar Shailubhai, PhD, whose drug TRULANCE was approved for treatment of chronic constipation in 2017 (3).

The Discovery Labs’ Innovation Hub in King of Prussia includes not only Unite IQ, the Center for Breakthrough Medicines, which offers research, development, and manufacturing services designed to provide companies of all sizes with better predictability and timeliness of medicinal delivery.

Scientific disciplines

BioPharm: Which areas and scientific disciplines need the most attention now and how will the incubator approach help?

Potts (Lonza): In our experience, manufacturability and scale-up, and the move from the lab to clinical or commercial production, are seldom as straightforward as emerging innovators believe they are. Working with an incubator can help by providing access to more manufacturing equipment-such as larger volume stirred tank reactors, or closed, automated systems such as Lonza’s Cocoon or Nucleofector platforms-at an affordable price, allowing early development to be improved so that process development may be smoother later on.

As large companies seed incubators, they should also consider providing sublicenses on a research-use basis to early stage innovators (often pre-funding or with minimal early funding) to allow them to access otherwise cost-prohibitive technology.

One area where this could be very useful is with exosome therapeutics, because many companies working in this space currently are pre-clinical and very early stage. By leveraging resources, companies working in this area could maximize speed to clinic.

Greenberg (Discovery Labs): CGT manufacturing is often a cumbersome and inefficient process that is difficult to produce at scale. The Unite IQ incubator approach adds value by allowing early-stage companies to adhere to a scalable platform process early in the product’s life cycle so that tech transfer can be seamless during the later stages of development when larger batches and more patient doses are needed.

Advantages

BioPharm: What advantages does the incubator approach provide to bridging R&D and production?

Greenberg (Discovery Labs): The Unite IQ incubator offers a full range of resources, talent, and services to expedite the path to commercialization by eliminating the typical bottlenecks, which include equipment, consumables, manufacturing capacity, talent, lab space, raw materials, and resources and providing them all in one place. The Unite IQ incubator model offers the full-scale lifecycle, including an innovative work environment and the platforms, processes, and capabilities that are needed to bring new discoveries to market. 

Potts (Lonza): Allowing early access to laboratory and manufacturing equipment and platforms, and a myriad of other products such as media formulations, cell and tissue acquisition services, can be a significant opportunity to leverage for CGT development. By directly developing their pre-clinical processes in manufacturing assets, innovators can streamline future process development and manufacturing scale up.

Moreover, by pooling resources with other companion companies in an incubator, companies may leverage incubator resources (e.g., small batch vector production for pre-clinical services). In some instances, incubators can enable their members by providing research evaluation agreements for enabling technologies like cell lines, plasmids, and vector.

Incubators allow the CGT industry to grow and to have healthy pipelines by allowing innovators to bridge the gap from early science to pre-clinical stages when the companies are typically spun-off, so that they will have raised enough cash to fund pre-IND activities and prep work. As these early stage resident companies scale up, the need to partner with good manufacturing practice (GMP) manufacturers such as Lonza, grows and further develops the therapeutic. Without this help, incubators would likely fall short in the area of GMP readiness as well as the process development required to get into GMP. Companies like Lonza can meet this need. While some contract development and manufacturing organizations (CDMOS) work with large companies in the CGT space, most of their customers are small biotechs with an academic research, rather than a commercial scale-up and manufacturing background.

BioPharm: How are intellectual property issues handled in the incubator model?

Potts (Lonza): Generally, there is no single ‘one-size-fits-all’ approach, but agreements are generally made so that they work in the best interest of both parties. Typically, the resident emerging company owns any intellectual property that it generates, based on inventorship laws that are also spelled out in a membership agreement. In the event that two resident companies are to formally collaborate on a given project, it is wise for them to enter into a formal collaboration agreement so they can clearly define and agree on how best to divide ownership of the technology between them.

The actual split can vary greatly. In some instances, academic institutions may retain a joint ownership or sole ownership of the intellectual property asset with a license back to the resident. Complicating the picture are country-specific laws and rules regarding inventor remuneration. Major markets such as the US do not have such provisions but other countries (e.g., Germany) do require inventor remuneration. Incubators must account for these local variations in their formation and in their member agreements.

As incubators continue to form and establish collaborative environments, determining intellectual property ownership and associated rights is crucial for any funding or contributing institutions and resident companies. Members must understand how to leverage these laws their best interest. Funding and contributing institutions, for example, may want to negotiate rights of first refusal in funding rounds or in licensing negotiations in order to ensure that they are able to recoup their investment of technology contribution.

Greenberg (Discovery Labs): The Unite IQ incubator has a robust screening process for prospective members and takes a three-pronged approach to protection and security: physical security, including access and segregation; electronic security, including firewalls and cyber policies; and human resources, and on-boarding security in the form of proper staff and contract employee screening.

References

1. Pennsylvania Biotechnology Center, “Pennsylvania Biotechnology Center Named One of Most Successful Life Sciences Incubators in United States,” Press Release, July 1, 2018.
2. Lasker Foundation, “Hepatitis C Replicon System and Drug Development,” Press Release, Sept. 12, 2016.
3. Baruch S. Blumberg Institute, “Baruch S. Blumberg Institute Honors Two Scientists for Drug Discoveries,” Press Release, March 2017.

Article Details

BioPharm International
Vol. 33, No. 6
June 2020
Pages: 46–48

Citation

When referring to this article, please cite it as L. Lavelle, "The Role of Incubators in Cell and Gene Therapy Development," BioPharm International 33 (6) 2020.