Empowering Gene Therapy Developers with Tailored Early Formulation Solutions

With more challenging molecules coming out of discovery every day, the early formulation stage of development can be a daunting time for gene - and cell therapy developers, but it doesn’t have to be that way.

Dr. Shaukat Ali, Sr. Director Scientific Affairs and Technical Marketing with Ascendia Pharmaceuticals shares his perspective to help walk clients through the process, highlighting the services that Ascendia can provide and some of the options available to suit the needs of their customers.

Solubility and Stability through tailored technologies

One of the challenges often faced during this early formulation stage of development revolves around molecule solubility, and Ascendia has the technology and the knowledge to help. Many marketed drugs face similar challenges with solubility, whether it be due to the molecule itself or one of the excipients in the formulation. Technologies offered by Ascendia such as their EmulSol®, AmorSol®, LipidSol™, and NanoSol® can be enabled in these situations to aid in the formulation of these poorly soluble molecules.

Customers can be confident knowing that Ascendia will perform full scale screening of their formulation to provide the right composition and find the right excipients in a uniquely tailored approach. Ascendia can work with customers to come up with unique, creative ways to handle these challenging molecules to develop prototype foundations for the client. Once the right formulations are identified, they can be scaled up not only to investigate their reproducibility, but also to find the solutions that are going to be the most stable and desirable. Many of these drugs not only have issues with solubility, but also with stability in formulation, and the technologies offered by Ascendia can assist in that as well.

The need for targeted drug delivery

Since gene therapy aims to target and repair damaged or abnormal genes, our LipidSol technology could be tailored - not only to fix these genes, but also to ensure the delivery of the therapeutic agent to their target gene or cell.

One such technology, based on lipid nanoparticles or LipidSol®, has gotten increased attention in the gene therapy industry in recent years as potential carriers for antibodies. Since antibodies are very large molecules with a high level of specificity for their target cells, care must be taken in the development of carrier molecules.

Using the lipid nanoparticle technologies provided by Ascendia, these antibodies can be attached to a carrier molecule to be delivered specifically to antigens on the target cell as a highly potent drug formulation, while avoiding off-target damage to normal cells.

Lipid nanoparticles as the new way forward: like draws like

Dr. Ali recounts how in his many years of experience, the biggest struggle in gene and cell therapy projects tends to be how gene actually transfects cells. A developer can have a really great formulation, but then these cells are just sitting together and unable to transfect.

This is a scenario that is experienced often with vaccines. If the lipids are not able to transfect the cells, the drug will just sit where it’s delivered.

Utilizing Ascendia’s novel lipid technology, the carefully selected lipids can transfect the cells, which can then transfect more cells, and so on and for forth, to result in the delivery of genes through systemic circulation. This propagation of transfected cells is the main draw to using lipid nanoparticles. Like draws like, and the membranes in the body are made of composition of different class of lipids. Being able to use these lipid nanoparticles to deliver exogenous particles that are capable of transfecting cells via endocytosis is really critical to the ability of these particles to penetrate and enter into the cell itself.

Ascendia- catering to the interests of the customer via tailored approaches

While there are other companies offering similar lipid-based technologies, Ascendia sets themselves apart with their customer focused approach. They have the ability to cater to both small and large customers through personalized, flexible approaches. Particularly with smaller customers, Ascendia can cater to their needs in ways that larger vendors may not be willing or able to.

Whether a customer is in the biotechnology sector, a small company, or even a virtual company, Ascendia works with them to understand their project, find the right solutions and determine how to best apply our technologies to provide a tailored solution.

Not only is Ascendia one of the top players in the field of lipid nanotechnology, but they also are committed to serving customers with any sized project. Currently, projects anywhere from pilot sized all the way to stage one testing can be accommodated with Ascendia’s facilities, with another facility currently being built. Starting in 2025, the production capacity will be increased into the hundreds of thousands range to better accommodate larger projects.

While production scale capabilities are increasing, Ascendia’s focus continues to remain on the premium valued by the customer.

Success at all stages

Companies worldwide can find success with Ascendia, building off of their conceptual work and proof of concepts, and letting us help them get their product formulated and produced for their trials. Ascendia has the manufacturing capacity to produce products following cGMP guidelines, with ISO clean rooms to ensure the sterility required for pharmaceutical manufacturing.

Additionally, Ascendia can aid in the transition from phase I clinical trials to phase II, and the drastic increase in supplies needed in such a transition in order to keep their preclinical studies moving forward.

Pioneering lipid technologies utilizing a robust toolbox

Ascendia is a pioneer in liposome technology, an invaluable asset to customers working with gene therapy. They can cater to the specific need of the customer, designing and creating tailored structures and liposomes for their target. Additionally, they can look into the drug design from a more holistic point of view, creating aggregates of structures to increase the drug loading, shelf life, and encapsulation efficiencies. Some of the most critical attributes of any delivery system are the adjuvants, which Ascendia can create using the best excipients and other components approved and listed in FDA’s inactive ingredient database (IID).

Ascendia has many tools in the toolbox, so to speak. They not only have excipients that have already been marketed, and have been granted GRAS status, but they also have libraries of novel excipients to work with. They can create the structures and the libraries to investigate gene encapsulation using nucleic acids, something made possible by the knowledge and experience provided by Ascendia.

Combining the best facilities with over a hundred years of experience in formulation, analytical support, and quality assurance, Ascendia can handle all of the challenges developers in the gene and cell therapy industry are facing. Customers can also expect to see more capabilities coming soon, with the construction of microbial labs, water for injection plant as Ascendia commits to taking their potential to the new heights. Contact Ascendia today!