By: Julianna LeMieux, PhD 

Next week, the members of the GEN team that focus on the work going on in the bioprocessing field are heading to Boston for the second time in as many months. This time, it will be to attend, and cover, the BioProcess International Meeting (BPI) where the bioprocessing community comes together to share ideas, learn about new equipment launches, network, and close deals with clients.

We were also in Beantown last month, to do the same, at the Bioprocessing Summit. One particularly inspiring talk from that meeting—a keynote given by Rachel Salzman, DVM, global head of corporate strategy at Armatus Bio—emphasized the importance of a sense of urgency when working to deliver gene therapy treatments to patients.

Salzman told a personal story of how both advocacy and urgency play key roles in gene therapies in her talk entitled, “Commercializing Gene Therapies—The Combined Power of Patient Advocacy and Cost-Effective Manufacturing.”

Salzman’s nephew was diagnosed with cerebral adrenoleukodystrophy (cALD), an X-linked metabolic disorder occurring in about 1 in 21,000 boys. cALD is also known as Lorenzo’s oil disease, based on the 1992 movie of the same name, in which Susan Sarandon and Nick Nolte play parents searching for a treatment for their son. The case of Salzman’s nephew started like many others of children with rare disease—with a diagnostic odyssey. The family was left with few treatment options; stem cell transplants are possible but require a suitable matched donor for the host (which FDA estimates is 30% of patients.)

The family came together to form the Stop ALD Foundation. After connecting with subject matter experts, they found that this disease was ripe for gene replacement therapy, which is what Salzman and her family set out to make happen.

The gene therapy for ALD (named eli-cel) was first developed by a company called Cell Genesys (which merged with BioSante in 2009) in the early 2000s. Salzman wove a story, spanning the past two decades, full of both roadblocks and successes. For example, a blow came in 2003, when the FDA halted all gene therapy trials that use retroviral vectors for delivery into bone marrow stem cells. The decision was a response to the news that a gene therapy for X-linked severe combined immune deficiency disorder (SCID) resulted in leukemia for two boys in a gene therapy trial in France. But there were hopeful milestones as well. For example, in 2006, gene therapy stopped the progression of ALD in two 7-year-old boys in a trial in France—the first time that a lentiviral vector treatment was ever used in humans who did not already have HIV infection. Bluebird Bio, founded in 2010, picked up the torch on eli-cel, filing an IND in 2013.

Salzman’s story ended on a hopeful note: the approval of Skysona by the FDA on September 16, 2022—the first and only approved gene therapy that slows the progression of neurologic dysfunction in boys with early, active cALD‑on.

However, the process took too long to save the life of her own nephew, who passed away in 2004. And, because Bluebird Bio closed their operations in Europe, citing financial reasons, boys in Europe have lost access to the drug. Salzman asserts that the cost of goods sold (COGS) have played a critical adverse role in these outcomes—not an uncommon sentiment from those in the bioprocessing world.

Salzman’s conclusions were less a summary of her talk and more of a call to action. First, she said, “the impossible is possible.” In her own experience, advocacy drove partnerships and alliances to advance science and technology that was otherwise siloed. Also, urgency leads to more rapid innovation. She added that the financial toxicity, and those related barriers, will increase over time. And, that COGS are a key contributor to this.

“There has to be a sense of urgency,” Salzman said, because urgency results in accelerated innovation timelines. (One need look no further than the quick progress made in COVID-19 for proof.) And, lastly, Salzman implored the audience to remember the patients: show their photos, talk to their families, and, above all, have the same sense of urgency that they do.

I will keep Salzman’s words in mind as I attend and cover BPI next week. Some of the gene therapy talks that I don’t want to miss will be the Tuesday morning keynote address by Tim Hunt, the CEO of the Alliance for Regenerative Medicine on the “State of the Industry and Future Perspectives for Cell and Gene Therapies.” Also, the talks by Rachel Legmann, the senior director of technology, gene therapy, Repligen and Ryan Bartock at Spark Therapeutics, who will speak on “Addressing the Challenges of Transferring and Manufacturing Gene Therapies Externally and At-Scale.”

Clive Glover, PhD, from Cytiva, will be presenting a talk titled, “Genomic Medicine Keeps Innovating. Manufacturing Should Too.” And, focusing on AAV, Yatindra Tirunagari from Rentschler Biopharma, will talk about “How Novel Tools Can Accelerate AAV Development Timelines.”

I am looking forward to learning, making connections, and continuing to cover the work going on in the field—all with a sense of urgency.

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