Latest news with #SparkTherapeutics
Boston Globe
19-05-2025
- Business
- Boston Globe
CRISPR pioneer Prime Medicine switches CEO and lays off quarter of its staff
Prime was initially formed in 2019, around a Get Starting Point A guide through the most important stories of the morning, delivered Monday through Friday. Enter Email Sign Up The technology can, in theory, be used to correct about 90 percent of known disease-causing mutations. It has revolutionized the field and proved so compelling that several competitors have been accused of copying the technology. But Prime itself had fallen on much harder times. Advertisement The company's stock is down 91 percent since its IPO. It had less than a year of cash left on its books. Last year, to conserve resources, it was forced to cut most of its pipeline of experimental treatments and conduct a small layoff. Outgoing CEO Keith Gottesdiener blamed investors that, with rising interest rates, Advertisement However, both outside executives and former employees also blamed Prime's leadership. Prime also faced delivery challenges, as scientists have not yet devised clear ways to send its gene editors anywhere in the body besides the liver. And the precision of the company's technology could actually work against it from a business standpoint. Many genetic diseases are caused by hundreds or thousands of different individual mutations. Changing one or two DNA letters at a time can mean treating only a small subset of an already rare disease. With Monday's announcement, Gottesdiener will be replaced by Allan Reine, a longtime biotech investor and Prime's CFO since January 2024. Prime said Gottesdiener decided to step down as CEO and a member of the board. The switch is 'effective immediately.' The company also named Jeff Marrazzo, former CEO of Spark Therapeutics, as executive chair. The reorganization will extend Prime's cash runway into 'the first half of 2026' and cut its expenses by half through 2027. As part of the move, the company is scrapping its lead program in one subtype of chronic granulomatous disease, or CGD, a rare immune deficiency that leaves patients vulnerable to life-threatening infections. The company had only managed to dose a single patient since receiving Food and Drug Administration clearance last April, an investigator announced at a gene therapy conference last week. That's likely because of the rarity of the condition and the complexity and intensity of the treatment: Cells have to be collected from a patient, edited in a lab, and then re-infused. Advertisement Prime said data from the patient were positive. After 30 days, 66 percent of neutrophils — a key immune cell impaired by the disease — had function restored. Only 20 percent correction is thought to be needed to benefit patients. It's a milestone, the first proof-of-concept for prime editing. But the company will cease all efforts to advance the program, as well as a follow-on therapy meant to help more CGD patients, likely because of the rarity of the disease and the difficulty other companies have had commercializing such complex treatments. Prime said it is working 'with urgency' to find another company who can take it forward 'to help ensure this important therapy is delivered to patients.' The company's main focus will now be on two liver conditions: Wilson's disease and alpha-1 antitrypsin deficiency, or AATD. Both are relatively common rare diseases. But both come with drawbacks. Although Wilson's disease can progress to liver failure and cause damage to other organ systems, some in the industry fear some patients may have their condition sufficiently well managed they wouldn't opt for a gene editing treatment. And in AATD, Prime has a Prime 'is committed to honoring the terms of the Agreement, and confident that it has the rights to pursue AATD under the Agreement,' the company said. Advertisement It is also competing with a slew of other biotechs developing both gene editing and non-gene editing treatments for the condition, in which a single letter misspelling leads patients' livers to secrete toxic proteins. In addition, Prime will continue work with the Cystic Fibrosis Foundation to create one-time therapies for the rare lung disorder and with Bristol Myers Squibb to create CAR-T treatments. Prime plans to put both its AATD and Wilson's disease programs in trials in 2026, meaning it will have to raise money before data from those efforts are in. It's not clear if any of its rivals are in a better position. The entire gene-editing industry's downturn has continued, and Tessera, the main company accused of copying Prime's technology, has stayed vague about its plans for starting trials.
Technical.ly
09-05-2025
- Business
- Technical.ly
Spark layoffs won't derail Philadelphia's biotech surge, city official says
Philly's life sciences sector is strategizing to remain at the forefront, despite recent shakeups at cell and gene therapy pioneer Spark Therapeutics. Turmoil at one big company doesn't necessarily indicate the end of a sector's strength, according to Rebecca Grant, senior director of life sciences and innovation for the city's Department of Commerce. While acknowledging the negative impact of job losses caused by the layoffs at Spark, Grant said she's still optimistic about the industry at large. 'People in these situations, they know how to pivot the innovation that they've created thus far,' Grant told reporter Sarah Huffman on Speaking, our monthly segment on WURD Radio's 'Reality Check' hosted by Tonya Pendleton. '[They] will help gene therapy and life sciences to move forward.' Earlier this year, Spark's parent company Roche announced a restructuring of the organization and 300 layoffs at its Philadelphia office. The abrupt shift — following Spark's major success story, from Penn spinout to the era's largest VC-backed exit — caused a moment of reflection for the cell and gene therapy scene. From Grant's perspective, it's been a time to recognize the moral issues at stake and the ecosystem's role in supporting these expensive endeavors. 'As these therapies come about that can actually cure people, and they don't have to continue to take drugs down the line, I think it takes all of us to get involved,' Grant said. Hopefully, jobs come with that. As a global company, Grant said Roche has the opportunity to bring more production jobs to Philadelphia. Those types of roles aren't just for scientists with advanced degrees. There are also training programs for 'aseptic techniques,' or keeping an area sterile, and biomanufacturing in the lab at local organizations like the Wistar Institute and the Skills Initiative. 'The research that's continuing,' Grant said, 'is how to make these treatments less expensive and how to manufacture them so that they are more affordable.'
Technical.ly
17-03-2025
- Business
- Technical.ly
Does the Spark Therapeutics writedown undermine Philly's biotech swagger? Startups have bigger things to worry about, they say
A shining star of Philadelphia's life sciences ecosystem recently faced a setback, putting a dent in the region's reputation for success in the sector. Cell and gene therapy standout Spark Therapeutics is being restructured following the end of its trial for a hemophilia A gene therapy treatment, and parent company Roche is classifying the former startup as a financial loss, according to the Philadelphia Inquirer. Spark says it's still working on a new gene product for the disease and stakeholders say this downturn doesn't discredit its years of impact. The situation is, however, calling attention to how life sciences startups face challenges like federal funding cuts and product development costs — though industry leaders still say they're optimistic about staying afloat by tapping into the talent and resources that exist here. One restructuring setback won't worsen investment opportunities for other life science ventures, according to Dean Miller, president of the Philadelphia Alliance for Capital and Technologies. 'You're not going to dissuade investors because a pharmaceutical company deprioritized an acquisition,' Miller said. 'That happens all the time.' Philadelphia is consistently a top region for venture capital and life sciences. The Philadelphia Metropolitan Statistical Area is a top-five venture capital market in the US, with life sciences as one of its major sectors, according to PitchBook's quarterly Venture Monitor report. Despite the high ranking, access to capital remains one of Philly's top challenges, especially locally, per Rebecca Grant, senior director of life sciences and innovation for the city's Department of Commerce. Recent challenges like Spark's signal that the industry needs to work toward making gene therapy manufacturing more cost effective, which would get more therapies to market and help attract investment, Grant told But it's also an example of how a company can pivot to stay afloat and continue working. 'They really created a lot of recognition for gene therapy and innovation,' Grant said. 'Now more people understand what gene therapy means and how it can literally cure disease.' Spark Therapeutics did not immediately respond to request for comment. Spark signaled strength in Philly life sciences — what does it mean now? Spark Therapeutics is one of Philly's go-to examples of success, and it helped build up the ecosystem in Philadelphia, Miller said. While restructuring could lead to staff cuts or shrinking offices, following layoffs at the company last summer, this is likely a short-term pain point, he said. After developing the first FDA-approved gene therapy, Luxturna, the Penn spinout broke into the mainstream. When pharma giant Roche acquired Spark in 2019, it was the largest VC-backed exit in Philadelphia at $4.8 billion, bringing more attention to Philly's life sciences ecosystem. '[It's] never easy when your trailblazer starts to disappear a little further.' Dean Miller, president of the Philadelphia Alliance for Capital and TechnologieS Since then, the company welcomed a new CEO and announced plans for its 500,000 square foot Gene Therapy Innovation Center in University City, which is expected to be completed next year. The company previously said the site would house over 500 jobs. 'Gene therapy is not a huge sector, and Spark was a trailblazer,' Miller said. '[It's] never easy when your trailblazer starts to disappear a little further.' Still, stories like Spark's are something that the ecosystem has seen before. Big successes come and go, but the momentum remains, he said, pointing to the example of Centocor, a biotech company founded in Philadelphia in 1979. Centocor's work was groundbreaking at the time, developing large molecule therapies and treatments for rheumatoid arthritis, Crohn's disease and other medical conditions, Miller said. Johnson and Johnson acquired Centocor in 1999 for $4.9 billion. At the time of acquisition, Centocor maintained its brand identity, but eventually completely integrated into Johnson and Johnson. However, the people who worked at Centocor are still around in the Philly life sciences ecosystem and helping new biotech startups get off the ground, Miller said. 'Twenty years from now, I think we'll be saying the same thing about Spark and its impact,' Miller said. 'Not just what it did in developing a new line and approach to gene therapy, but how its people went on to found other companies, to fund other companies.' Fed funding blocks, fleeing investors are the bigger rift Current economic uncertainty and funding changes at the federal level do signal a bit more concern around Spark's turmoil, Miller said. The Trump Administration recently proposed huge cuts to funding for the National Institutes of Health, already seeing grants for vaccine-hesitancy research being rescinded. Philadelphia received $5.9 billion from the NIH between 2019 and 2023, according to the Commerce Department's 2024 Life Sciences Impact Report. Without NIH funding, the United States will fall behind in terms of medical research, Grant said. While stakeholders hold out hope that the administration will change course, the ecosystem needs to start thinking about alternative funding opportunities, like philanthropic foundations and a local pilot program that Grant said the city is working on to target early-stage life sciences companies through the city's small business catalyst fund. 'It takes millions and millions of pre-revenue dollars to bring a drug to market,' Grant said. 'So I'm developing and thinking out a program that I hope to release soon that can help small companies around the city that are moving towards commercialization.' However, this uncertainty could offer a benefit for Philly, she said. If there are fewer funding opportunities and companies have to be more careful with money, Philadelphia's affordability could attract more companies. This could also be an opportunity to increase collaboration across the ecosystem and make capital stretch farther, she said. Researchers doing similar work at different institutions may choose to team up and have a better shot at funding while they wait for more VCs to come through with cash. 'We would always love to see some bigger, deeper-pocketed investors,' said Kathie Jordan, managing director of the healthcare investment group at Ben Franklin Technology Partners of Southeastern Pennsylvania, 'and we continue to work on building out those relationships with investors who really can lead those rounds in the tens of millions.' Philly leans on new labs, strong workforce to push ahead Despite setbacks, Philadelphia continues to lean into its strengths and work toward growing the life sciences ecosystem. Lab and office space is a huge opportunity in the region right now, Grant from the Commerce Department said. Developers are continuing to build more workspace and companies are showing interest in these spaces even before they're finished. 'I don't think people would be continuing to invest if they felt like this ecosystem was going to fail,' she said. 'I don't think people would be continuing to invest if they felt like this ecosystem was going to fail.' Rebecca Grant, senior director of life sciences and innovation for Philly's Department of Commerce The city also has a strong talent pool and workforce, she said. The region ranked No. 8 for life sciences talent on CBRE's 2024 US Life Sciences Talent Trends report, slipping down two spots, but remaining in the top 10 for the third year in a row. The lower cost of living and proximity of major research institutions are also benefits. New success stories are also coming up. For example, Mineralys Therapeutics stock price surged after announcing positive clinical trial results for its hypertension drug candidate lorundrostat. The company raised $192 million after going public last year. As companies like Mineralys continue to grow, they will also hire the talent that exists here in the market, Miller said. But Philly needs to learn how to market itself better and get comfortable touting its accomplishments, Grant said. The key is to 'evangelize' everything the ecosystem has to offer. 'We've always maintained our head above water,' Grant said. 'The innovation we're creating here is just so important to the greater good globally. We'll continue to innovate.' Sarah Huffman is a 2022-2024 corps member for Report for America, an initiative of The Groundtruth Project that pairs young journalists with local newsrooms. This position is supported by the Lenfest Institute for Journalism.
