Latest news with #hemophiliaA
Daily Mail
13-05-2025
- Health
- Daily Mail
DC man suffers terrifying rare bleeding disorder after Covid vaccine booster
A Washington DC man was left excessively bleeding due to a rare side effect linked to his Covid vaccine booster. The unidentified man who is in his 70s went to the hospital after he saw blood in his stool - believing it was due to the colonoscopy he had the day before. But while examining him, doctors also noticed large, dark bruises on both of his arms that had developed within the span of a few days. Tests revealed he was suffering from hemophilia A, where the body prevents blood from clotting, causing excessive bleeding from wounds for days at end. This prolonged bleeding can cause blood to accumulate beneath the skin, forming a bruise and make the body more susceptible to injuries. In this particular case, since the patient had sustained wounds from a colorectal procedure, they were mostly bleeding internally and the blood was only visible in their stool. The patient had no family history of the condition and was not actively bleeding, according to a medical report about his case. However, they traced his symptoms back a week after the elderly patient had received his Moderna Covid vaccine booster - marking it as his fifth shot. The experts theorized that it was possible the shot triggered his immune system to produce antibodies against his body's clotting process and caused the disorder. Typically, mRNA vaccines work by prompting the immune system to produce SARS-CoV-2 spike proteins to attack the Covid virus in the body through a piece of genetic code. However, in this case doctors believe that it may be possible that the vaccine also made the body stop recognizing its own cells as safe and began developing antibodies against the clotting process. Experts have noted that adverse reactions to Covid vaccines are extremely rare and that the benefits of getting the shot outweigh potential complications After his diagnosis, the experts reviewed 21 cases of AHA developing in patients after receiving Covid vaccines between 2020 to 2022 and discovered that they usually were in their 70s and symptoms tend to show 14 days after receiving the shot. They also noted that the condition can be fatal in about 10.3 percent of cases which is why early diagnosis and treatment are crucial for survival. AHA can cause bleeding in the skin, muscles, and soft tissues but more concerningly, it can also lead to internal bleeding, including in the brain and gastrointestinal tract - which if not controlled, can cause the body to start bleeding out and in certain cases, lead to death. It remains unclear exactly how the vaccine triggered specific clotting antibody production in the patients' bodies. Most cases OF AHA occurred after people received their first or second dose of the Covid vaccine - when the immune response is highest. However, this particular man developed the bleeding disorder after his fifth dose - making his case distinct. When the elderly man initially went to the doctors, his white blood cell count, platelets count, liver and kidney function all appeared normal during tests. But much to their surprise, the bruising on his arms continued to worsen and he developed large marks on his left knee two weeks later - prompting doctors to order more intensive blood tests which included a plasma test. Results showed that while his white cell and platelets counts were normal, they were certain antibodies present in his plasma that were not allowing the body's clotting process to function. Further testing showed that his body's FVIII, crucial protein involved in forming blood clots to stop bleeding, levels were only at one percent in his body. As a result, he was diagnosed with acquired hemophilia A - which is known to primarily target the FVIII protein in the body. Typical symptoms of this condition include nosebleeds, bruising throughout the body, solid swellings of congealed blood, blood in the urine and gastrointestinal bleeding. Consequently, patients also develop also complications associated with abnormal, uncontrolled bleeding into the muscles, skin and soft tissue that can occur spontaneously, during surgery or following trauma. While it is usually treatable, AHA can potentially cause life-threatening bleeding complications in certain cases. Experts estimate that the condition affects about two people per a million in the US - meaning that it is diagnosed in nearly 1,000 people in a population of 340 million people. As for treatment, the elderly man was asked to visit a haematology clinic every week and began taking cyclophosphamide (an immunosuppressant medication used to treat autoimmune diseases). The doctors progressively decreased his cyclophosphamide dosage as his FVIII levels improved and he showed signs of recovery. Along with this, he was also given medications to prevent fungal infections and protect the stomach.
Technical.ly
04-04-2025
- Business
- Technical.ly
Spark Therapeutics files notice to lay off 300 employees this year
Cell and gene therapy standout Spark Therapeutics is undergoing its second shakeup in a month, with plans to lay off about half of its workforce. Several hundred people will be affected. A WARN notice, which companies file to provide advance notice of layoffs, reported 298 eliminated positions in the Philadelphia region. A Spark Therapeutics spokesperson told the Philadelphia Business Journal on Thursday it would be laying off 337 of its almost 650 employees. These changes are expected to occur in three waves: in May, July and at the end of 2025. The remaining 310 employees will be incorporated into parent company Roche, a multinational pharmaceutical company. Spark first announced on January 30 the decision to integrate more of its work into Roche, spokesperson Denise Bradley told The impacted employees will be eligible for severance, outplacement services and will be able to apply for other roles within Roche. Spark's plans for its University City-based Gene Therapy Innovation Center in Philadelphia have not changed, Bradley said. Last month, Roche classified the former startup as a financial loss following the end of its trial for a hemophilia A gene therapy treatment, the Philadelphia Inquirer reported. The company is still working on a new Hemophilia A gene product, Spark previously told The layoff announcement comes less than a year after Spark's previous workforce reduction, when it let less than 50 of its employees go in July 2024. At the end of last year, the company welcomed Roche veteran Sylke Poehling as its new CEO, replacing Ron Phillip, who had been in the role since 2022. Spark's year of downsizing Recent struggles at Spark, which was founded by Jeffrey Marrazzo in 2013 and was considered a big Philadelphia success story, indicate the need for the gene therapy sector to make manufacturing more cost effective, Rebecca Grant, senior director of life sciences and innovation for the city's Department of Commerce, previously told But the company itself heavily contributed to the development of the industry as a whole. '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.' In 2021, the company announced plans for a 500,000-square-foot Gene Therapy Innovation Center in University City. At the time, Spark said the new site would house over 500 jobs. The Innovation Center is still expected to be completed next year, Spark spokesperson Bradley previously told The Penn spinout is known for developing the first FDA-approved gene therapy, Luxturna. Pharma giant Roche acquired Spark in 2019 for $4.8 billion, the largest VC-backed exit in Philadelphia at the time. 'Gene therapy is not a huge sector, and Spark was a trailblazer,' Dean Miller, president of the Philadelphia Alliance for Capital and Technologies, previously said. '[It's] never easy when your trailblazer starts to disappear a little further.' 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.
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.
