Tough Road Ahead For Bluebird’s Gene Therapy


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Last Friday marked a biotech milestone as the Food and Drug Administration approved two gene therapies for the treatment of sickle cell disease. The first was Casgevy, which was co-developed by Crispr Therapeutics and Vertex Pharmaceuticals—this is the first gene therapy approved that utilized Crispr-based technology. The second was Lyfgenia, developed by Bluebird Bio. Bluebird also presented promising data about its drug at the American Society of Hematology annual meeting over the weekend, which suggested 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 Lyfgenia at $3.1 million, compared to the $2.2 million price tag that Vertex placed on Casgevy. 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 Wedbush 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 Andrew Obenshain 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.”

Correction: A previous version of this article erroneously said that Bluebird’s treatment also utilized Crispr.

Supreme Court Agrees To Hear Abortion Pill Cases

The Supreme Court will determine to what extent abortion pills should be restricted as it agreed this week to take up two cases concerning the legality of mifepristone. The move comes two years after the conservative-leaning high court overturned Roe v. Wade.

Read more here.

Pipeline & Deal Updates

Stealth Emergence: 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 a $4.3 million seed funding round. Another is Portal Bio, which is developing a cell engineering platform for a multitude of different therapeutic types. Finally there’s Tome Biosciences, a gene editing startup that emerged from stealth having already raised over $200 million in series A and series B rounds.

Crispr License: Vertex Pharmaceuticals announced it’s entering into a $200 million non-exclusive license with Editas Medicine for use of that company’s Cas9 gene editing technology in its sickle cell treatment Casvegy.

Cell Encapsulation: Encellin, which is developing a platform that enhances the durability of therapeutic cells, announced it has raised a $9.9 million round led by Khosla Ventures.

Reproductive Immunotherapy: Freya Biosciences, which is developing immunotherapies targeting vaginal microbiota to treat infertility, announced it has raised a $38 million series A round led by OMX Ventures and Sofinnova Partners.

Oncology: Bicara Therapeutics, which is developing biologic therapies for multiple cancer types, announced it has raised a $165 million series C round.

Radiopharma: Artbio, which is developing radioligand therapies, announced it has closed a $90 million series A round co-led by Third Rock Ventures and an undisclosed firm.

Synthetic Bio: The Chan Zuckerberg Initiative, Allen Institute and University of Washington have launched 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.

Generative AI: Google released MedLM today, a “ family of foundation models fine-tuned for the healthcare industry.” Early customers include HCA Healthcare and Augmedix working on ambient note-taking and BenchSci for drug development R&D.

SmileDirectClub’s Abrupt Shutdown

Dental aligner company SmileDirectClub shut down operations last Friday, leaving patients confused about the future of their dental health—and their payment plans. The company advised customers to seek future care from local dentists, continue making payments for their treatments and said the status of refunds is still in limbo.

Read more here.

Other Healthcare News

Billionaires Ken Griffin and David Geffen are donating $400 million to New York-based Memorial Sloan Kettering Cancer Center.

AstraZeneca will acquire RSV vaccine developer Icosavax for $1.1 billion.

Shares of Pfizer dropped to their lowest level since before the Covid-19 pandemic, as the company’s revenue estimates fell below analyst targets.

Health insurer Cigna is planning $10 billion in stock buybacks as it scraps Humana merger.

A new study found patients using Eli Lilly’s weight-loss drug Zepbound experienced a “substantial regain” of lost weight after stopping treatment.

A pregnant woman in Kentucky is challenging the state’s strict six-week abortion ban with a class action lawsuit. A woman in Texas who sued over a similarly restrictive law was halted by the state Supreme Court and left the state to get the procedure.

Grammy-award nominated singer-songwriter Aloe Blacc talks to Forbes about his biotech company Major, which is developing treatments for infectious disease.

Across Forbes

Beyoncé Is Now Worth $800 Million

Will Former NYSE Boss Tom Farley Be The Next Crypto King?

Exclusive: Mars Unveils Strategy To Double Snacking Revenue To $36 Billion

What Else We are Reading

Millions in Opioid Settlement Funds Sit Untouched as Overdose Deaths Rise (KFF Health News)

Is the flu shot market a slam dunk for mRNA vaccines? Experts aren’t so sure (STAT)

More than 10,000 research papers were retracted in 2023 — a new record (Nature)


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