“We’re investing in what companies will be doing in the future,” Burns said of the company’s focus on developing new treatment approaches.
Unlike many biotech startup companies, Casebia Therapeutics has had the good fortune to not have to worry about fundraising efforts to finance its gene-editing development programs.
Casebia was formed in 2016 as a joint venture between pharma giant Bayer and CRISPR Therapeutics with an aim to use gene-editing techniques to develop treatments for blood disorders and autoimmune, metabolic and congenital heart diseases.
Sanofi veteran Jim Burns, Casebia’s chief executive officer, told BioSpace that Germany-based Bayer wanted to become more heavily involved with innovation and game-changing technology, which was why it backed the cutting-edge techniques of CRISPR.
In the past three years, Casebia has ramped up hiring to support its scientific research, which is beginning to pay off. Burns pointed to April’s American Society of Gene & Cell Therapy conference that featured 10 different presentations made by Casebia, four of which were oral presentations. The bang the company made at the conference was what Burns considers to be the company emerging from stealth mode.
“We’re investing in what companies will be doing in the future,” Burns said of the company’s focus on developing new treatment approaches. As an example, he pointed to Casebia’s synthetic RNA-guided nucleases designated Cas-sRGNs. While more people are familiar with the Cas9 used in gene editing, he said that protein was not designed specifically for gene editing. The sRGN protein is. Burns said the company’s sRGN protein is synthetic, which makes it less susceptible to an attack from the body’s immune system. The Cas9 is bacterial in nature, so the body could turn on it, Burns said.
“These are next generation nucleuses and we’re pretty far ahead on these,” Burns said.
Not only is Casebia developing these next-generation sRGN delivery methods for gene editing, but Burns said the company’s AAVs are also capable of self-inactivation. Casebia is developing an Adeno Viral Vector that “makes the protein, does its job and stops making the protein.” This could enhance the safety profile of the treatment by reducing potential immunogenicity and off-target effects, he said. Currently, the company is aiming this as a new way of treating retinitis pigmentosa, a rare genetic disorder that involves a breakdown and loss of cells in the retina. However, that work is still in the early stages.
“If you have a self-limiting approach, it does reduce the chances of unintended consequences with CRISPR,” Burns said.
While gene editing has a near-seeming limitless approach to treating certain rare disorders, there are also serious ethical concerns at play in the field, highlighted by embryonic research conducted in China, which BioSpace has covered on multiple occasions. While that action has rightly earned worldwide condemnation, Burns noted that it opened up the conversation to provide platforms for companies like Casebia to show the therapies they are trying to develop to help people. Burns said on the company website there is the proclamation of a policy that the company only works with somatic cells, cells that are in adults, not embryos. The policy had been in place prior to the Chinese experiment, Burns added.
The company is eying the opportunity to take its first two therapies into the clinic. Casebia is in the process of seeking approval to take its therapy for IPEX (Immune dysregulation, polyendocrinopathy, enteropathy, X-linked) syndrome into the clinic next year. IPEX syndrome, which typically affects young children, is characterized by the development of multiple autoimmune disorders in affected individuals. Burns said the hope is the company will be able to engineer regulatory T cells and insert them into the patients as a means of treating the autoimmune disorder. In addition to IPEX syndrome, Casebia plans to tackle another undisclosed autoimmune indication with an IND filing. Burns said the company is not ready to publicly release information regarding this particular indication.
Until the filing of those INDs, Burns said the next year will likely include the publication and presentation of additional scientific research papers, as well as a continued building out of the company. Burns said the company currently has 89 employees and will likely expand to about 120 employees as it gets ready for the clinic. The company recently participated in Project Onramp, which provides paid life sciences internships for students from low-income backgrounds.
“We are developing a broad and deep toolbox of engineering tools,” Burns said.
