Jang-Ho Cha had no interest in leaving his post at Novartis, where he led translational medicine for the pharma giant’s work in neuroscience.
But in the summer of 2022, he received a call from a leader in the field of gene therapy: Beverly Davidson, co-founder of Spark Therapeutics and director of the Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia. She formed a biotech based on exciting vector capsid work coming out of her lab and wanted to see if Cha would join. The two had been friends for two decades, and given how much he admired her scientific rigor, he decided to listen to her pitch.
They met at a conference and discussed the CNS gene therapy capsids that the company was developing. The two talked over lunch at a Thai restaurant, and he was immediately sold.
“What she was showing me was a sea of green glowing neurons in the non-human primate brain that were completely transduced,” Cha said in an exclusive interview with Endpoints News. “To me, it was like somebody had just opened up this box and you recognize, ‘Well, whoa, that’s a dragon’s egg. This is incredible.’”
“Now I have to quit this job that I really like,” Cha recalled of his thinking at the time. “I have to go do this thing.”
He would become chief scientific officer of the startup, Latus Bio, in December 2022.
On Thursday, the Philadelphia biotech emerged with $54 million in initial Series A financing that will help Latus expand to Boston in June and enter human studies in late 2025, Cha told Endpoints. The company’s pipeline includes gene therapies for CLN2, Huntington’s disease and a third undisclosed indication.
8VC and DCVC Bio led the round, which could increase to about $75 million to $90 million if more investors come in, Cha said. Additional backers include Samsung Bioepis, Samsung Life Science Fund, the Children’s Hospital of Philadelphia Foundation, Benjamin Franklin Technology Partners, Modi Ventures and Gaingels.
Steering the startup is chief executive Peter Ghoroghchian, who previously led Ceptur Therapeutics.
“It’s a little ironic that a trained and former practicing medical oncologist who’s a non-viral gene therapy guy ends up as a CEO of a CNS viral gene therapy company,” Ghoroghchian said. “But it all comes to the state of the science, and especially the tropisms of the novel capsid variants that Latus has for specific substructures and cell types in the CNS. I have never seen the specificity of a delivery system such as what Latus has.”
Spark to Latus
Davidson is trying to replicate the success she and the Spark team had. The startup became a fixture in the gene therapy space, snagging one of the field’s first FDA green lights for its inherited vision loss treatment Luxturna in 2017 before selling to Roche for $4.3 billion in 2019.
In the years following Spark, Davidson and her team at CHOP discovered new, highly potent capsids, a key component of delivering AAV gene therapies.
“Some groups got wind of this,” Davidson said, and requests started coming in for their capsids. It was time to create a company around them, she decided. Their wide potential led to the name Latus, which is Latin for “broad,” Davidson said.
The goal is to create one-and-done gene therapies that don’t expose the liver, spleen or kidney, and avoid systemic toxicity, Davidson and Cha said. The gene therapies will be delivered directly to the brain at low doses, Cha said. The startup expects its treatments to be part of outpatient procedures done with under two hours of anesthesia, Ghoroghchian said.
Kiersten Stead“Because our vectors are so potent, it reduces the cost of goods, which addresses something that you’ve heard over and again from [the FDA’s] Peter Marks in that we have to make the scalability of these transformative therapies real, and we’ve certainly taken a very large first step in making that happen,” Davidson told Endpoints.
“When you have a vector that’s 100 times more potent, and you run the same manufacturing process, instead of treating 10 patients, now you have enough product to treat 1,000 patients,” she continued.
The potency is one of the attributes that attracted investor DCVC Bio, said Kiersten Stead, managing partner at the firm, whose investments include Umoja Biopharma and Solu Therapeutics, among others. DCVC Bio had stayed away from earlier versions of AAV gene therapies because of potency and other manufacturing bottlenecks, she said. DCVC, meanwhile, has invested in biotechs, as well, including AI-driven Relation Therapeutics.
Pipeline built on decades of interest
Latus plans to enter the clinic before the end of 2025. Its first test will be in patients with neuronal ceroid lipofuscinosis type 2 disease, or CLN2, which is a form of Batten disease that progresses quickly and can lead to seizures, vision loss and motor impairments, among other symptoms.
BioMarin secured the first FDA approval for treating CLN2 in 2017, but the medicine can be burdensome for children, who have to go into infusion centers every few weeks. Known as Brineura, the enzyme replacement therapy reeled in $39 million in net revenue in the first three months of 2024, the same amount as the first quarter of 2023.
Latus hopes its one-time gene therapy will “hit high therapeutic levels of the missing enzyme,” known as TPP1, Cha said.
Other gene therapy companies have also explored treatments for forms of CLN2, but Lexeo Therapeutics and Regenxbio now seek partners for their candidates.
After CLN2, Latus hopes to test a gene therapy in patients with Huntington’s disease, in which nerve cells in the brain dwindle, causing disruptions to movement, thinking and other functions. The company is going after two targets, MSH3 and PIAS1, which are “key accomplices in how mutant huntingtin causes cellular damage,” Cha said.
Huntington’s disease is an area of particular interest for Cha and Davidson. The two have worked in the field for decades. As a medical school student, Cha was part of a research project in Venezuela that gave researchers key insights into the disease’s genetic background.
“In my heart, we’ve always been focused on the patient. I don’t create these vectors just to create them and then say, ‘Where do they go and how can I apply them?’” Davidson said. “We really started with the patient in mind first and the disease indication first and work backward.”
Multiple other biotechs are already testing, or plan to develop, gene therapies for the inherited disorder, including uniQure, Bayer, Voyager and Roche’s Spark.
Davidson plans to share more about Latus during her keynote speech at next week’s annual confab of the American Society of Gene & Cell Therapy in Baltimore.
Editor’s note: This story was updated to correct that DCVC, not DCVC Bio, invested in Relation Therapeutics.