InnovationRx: Tough Road Ahead For Bluebird’s Gene Therapy

Last Friday marked a biotech milestone as the approved two Cripsr-based gene therapies for the treatment of sickle cell disease. The first , which was co-developed by and . The , developed by .

Bluebird also presented promising data about its drug at the American Society of Hematology annual meeting over the weekend, a durable response to the treatment for sickle cell patients five years after receiving treatment. Despite the approval, though, Bluebird finds itself in a tough position compared to its competitor. For one, it released pricing for at $3.

1 million, compared to the $2. 2 million price tag that Vertex placed on . Second, the FDA approval for Lyfgenia includes a safety warning that requires patients to be monitored for a potential cancer risk, something that Casgevy’s approval does not.

Additionally, Bluebird had expected to receive a priority review voucher (PRV) worth about $103 million following approval, but the FDA didn’t grant it. These PRVs aren’t money, but rather a guarantee from the FDA that it will speed up review of a future drug developed by a company because it chose to develop a drug for a rare disease. The FDA allows PRVs to be sold to other companies, which enhances their value.

This combination of factors caused the stock price to drop nearly 40% on Friday. The company only has about enough cash runway right now to last through the second quarter of 2024, leading analysts at to conclude that “[w]ithout access to significant amounts of capital to support Lyfgenia’s launch and the competition from Casgevy, we question the viability of the Lyfgenia franchise. ” Unsurprisingly, Bluebird CEO disagrees with this assessment, telling Forbes that Bluebird will appeal the FDA’s decision to not grant the PRV, but that it was never the only way that the company intended to obtain its needed capital.

“The PRV was always one component of our plan, so we just plan to execute the other components,” he said. He declined to go into specifics though did highlight the fact that Bluebird already has two other gene therapies on the market generating revenue. Those two therapies give Bluebird another advantage going into 2024: the company already has the treatment and support it needs to bring Lyfgenia to market.

“We’ve already built the infrastructure we need,” said Obenshain. This not only means the company needs less capital to go to market, but also offers a market differentiator, because it already has experience in patient side operations for gene therapy. Ultimately, Obenshain said, “We feel at Bluebird we both have a mission both to deliver these therapies but also to show that a standalone cell and gene therapy can make it,” adding that “if we can’t make it, I think that does not bode well for innovation.

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Wade. Pipeline & Deal Updates Three interesting startups emerged from stealth this week. One is Inductive Bio, which is developing a machine learning platform to optimize compounds in drug discovery, emerging with .

Another is Portal Bio, which is developing a for a multitude of different therapeutic types. Finally there’s Tome Biosciences, a gene editing startup that emerged from stealth having already in series A and series B rounds. Vertex Pharmaceuticals announced it’s entering into with Editas Medicine for use of that company’s Cas9 gene editing technology in its sickle cell treatment Casvegy.

Encellin, which is developing a platform that enhances the durability of therapeutic cells, announced it has led by Khosla Ventures. Freya Biosciences, which is developing immunotherapies targeting vaginal microbiota to treat infertility, announced it has raised led by OMX Ventures and Sofinnova Partners. Bicara Therapeutics, which is developing biologic therapies for multiple cancer types, announced it has raised .

Artbio, which is developing radioligand therapies, announced it has closed co-led by Third Rock Ventures and an undisclosed firm. The Chan Zuckerberg Initiative, Allen Institute and University of Washington the Seattle Hub for Synthetic Biology, which aims to, among other things, develop technologies that enable the measurements of cells over the course of their lifespans in order to bolster biological research. Google MedLM today, a “ family of foundation models fine-tuned for the healthcare industry.

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