Companies Enter Licensing Agreements to Expand Gene Therapy Programs

Gene Therapy

Days after forging a $1.1 billion agreement with Selecta Biosciences to develop gene therapies for lysosomal storage disorders, Takeda Pharmaceutical entered into a licensing pact with Poseida Therapeutics for its Cas-CLOVER nanoparticle delivery technology and other genetic engineering platforms. The deal is worth up to $3.6 billion

Takeda intends to use Poseida’s proprietary technology to research and develop up to eight gene therapies. According to the announcement, the collaboration will focus on the development of non-viral in vivo gene therapy programs, including Poseida's Hemophilia A program. 

The partnership will initially focus on developing six in vivo gene therapy programs that harness Poseida's novel technology platforms. Takeda can also add two additional programs to the collaboration and is bound to grant funding for all collaboration program R&D costs.

Madhu Natarajan, head of Takeda’s Rare Diseases Drug Discovery Unit, explained that Poseida’s differentiated platform technologies “show great promise in developing non-viral in vivo gene therapies.” The partnership also reinforces Takeda’s commitment to developing next-generation gene therapies for patients with rare genetic and hematologic diseases.

Under terms of the deal, Takeda paid San Diego-based Poseida $45 million upfront. When milestones are factored in for all eight programs, the partnership could be worth up to $3.6 billion. Poseida will lead the research activities through candidate selection. Following that, Takeda will assume responsibility for further development and commercialization.

For Takeda, this follows the Selecta partnership that aims to leverage that company’s ImmTOR platform that could potentially allow for re-dosing of gene therapies. The platform is intended to lessen unwanted immune responses, allowing for multiple dosing and longer responses.

Takeda wasn’t the only company to expand gene therapy programs. Spark Therapeutics, a Roche subsidiary, entered into a licensing agreement with CombiGene AB for that company’s CG01 project. CombiGene’s CG01 project is an investigational gene therapy that aims to treat drug resistant focal epilepsy. CG01 is in a late preclinical stage but could potentially treat a large patient population. 

Federico Mingozzi, chief scientific officer at Spark Therapeutics, noted that a population of epilepsy patients "are in need of new therapeutic options" as they do not respond to current anti-seizure therapies. The Philadelphia-based company, which successfully developed a gene therapy for a rare form of blindness, intends to turn its expertise "in gene therapy to address unmet needs for people living with drug-resistant focal epilepsy."

Jan Nilsson, chief executive officer of CombiGene, affirmed that CG01 has continued to show strength throughout its preclinical development. He also showed confidence that Spark is the perfect partner to take CG01 into clinical development and potential commercialization. 

“CombiGene and Spark have had a productive ongoing dialogue during the latter parts of CG01’s preclinical development, and the entire CombiGene team have come to know Spark as a visionary and patient-focused organization with the strength, know-how, and experience to exploit the full potential of CG01. We look forward to advancing this potentially transformative therapy together with Spark for the benefit of a patient group in need of better treatments,” Nilsson said in a statement.

Financial terms of the agreement were not disclosed. 

Also, California-based BridgeBio Pharma announced plans for its second annual R&D Day that will showcase some of the company’s gene therapy programs, as well as its KRAS inhibitors for cancer. Gene therapy programs that will be highlighted at the R&D Day include BBP-818, a new adeno-associated virus (AAV) gene therapy program for classic galactosemia (severe GALT deficiency).

Classic galactosemia is caused by a severe deficiency of the enzyme galactose-1-phosphate uridylyltransferase (GALT). BBP-818 is designed to enable the GALT enzyme production and enable the body's natural ability to metabolize galactose. Preclinical studies in a mouse model of classic galactosemia showed that BBP-818 restored up to 72% of wild-type levels of GALT enzyme in the brain following a single dose.

In addition to its GALT program, BridgeBio is advancing gene therapy candidates for Canavan disease and congenital adrenal hyperplasia (CAH). The company also has a preclinical program for TMC1 hearing loss. 

BridgeBio will also announce preclinical gene therapy programs targeting tuberous sclerosis, cystinuria and a genetic dilated cardiomyopathy.

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