Starting fresh in manufacturing, Forbion startup refuels to steer next-gen gene therapy approach for ALS, Alzheimer's into clinic
Forbion laid out its case for a next-generation gene therapy approach when it took the wraps off VectorY Therapeutics and its vectorized antibody tech in February. Now, the Dutch VC has tapped an experienced hand at cell and gene therapy manufacturing to steer the ship — and pulled a marquee syndicate for a €31 million ($37.6 million) seed round.
Alexander Vos, the new CEO, is a venture partner at BioGeneration Ventures and jumps immediately from VarmX, a BGV portfolio company developing an anticoagulant. But before that, he had led Dutch CDMO PharmaCell for eight years until it was bought out by Lonza.
There, Vos got a close-up look at the limits of the traditional gene replacement approach and viral vector manufacturing, from lack of specificity for certain tissues to low efficiency. He had seen companies walk in with semi-academic processes that would take a long time to scale up.
That’s just not going to cut it these days.
“If we are going to address larger disease areas in the CNS and muscle disorder space, we have to bring down the cost of goods and we have to get better at manufacturing,” he said.
Those are the two disease areas that VectorY is pursuing, with the hypothesis that if you could just get past the classic distribution barriers and express an antibody inside cells through a vector that encodes for it, you can make a real difference in diseases like amyotrophic lateral sclerosis, Alzheimer’s disease and a variety of muscle disorders.
In proving that, they will also have to sidestep the safety issues that have troubled Voyager’s vectorized antibody programs in similar neuro diseases including ALS, Parkinson’s and Alzheimer’s.
For ALS in particular, VectorY has teamed up with an unnamed partner to vectorize their antibody candidates, an approach that they believe will be more effective because it can get into the brain better.
With animal studies now ongoing, Vos notes the ALS program will likely become the lead program with clinical studies on the horizon for 2023.
They will pursue a similar idea in Alzheimer’s — a field that’s now changed psychologically, Vos remarked, because the approval of Biogen’s Aduhelm showed it’s possible — in terms of vectorizing antibodies that can clear protein aggregates, although the exact approach has not been decided. The first muscle indication also remains to be selected, which will likely happen later this year.
“We see our pipeline expanding through multiple collaborations combining worldclass antibody knowhow with our knowhow on the gene therapy and vectorized antibody space,” he added.
Joining the group of gene therapy vets already on the team will be Sander van Deventer, co-founder of Forbion, who is perhaps best known for his work on the gene therapy Glybera and then helping create uniQure — a place both CSO Pavlina Konstantinova and VP of vector development Barbara Sanders have spent time at. Sanofi Genzyme vet Carlo Incerti continues to chair the board while Marco Boorsma, formerly interim CEO, will now step back and take a board seat.
Building an in-house manufacturing capacity with this experienced crew is key to Vos’ plan for the company, initially with a CDMO partner whom it will help on the process development front in exchange for a dedicated part of the facility, so that they won’t be at the mercy of scheduling.
In the long term, the plan — backed by Forbion and new investors at BGV as well as Eli Lilly — will be to establish its own plant in the Netherlands.
“The truth always is that you have enormous latency in the industry, because people are locked into their processes because of the CMC and regulatory environment, right? They cannot change away from that. So it’s always slow,” he said. “I think it’s sometimes easier to just start fresh.”