Latest news with #CRISPR-based
Business Insider
3 days ago
- Business
- Business Insider
CRISPR Therapeutics to Participate in Upcoming Investor Conferences
ZUG, Switzerland and BOSTON, May 29, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that members of its senior management team are scheduled to participate in the following investor conferences in June. William Blair's 45 th Annual Growth Stock Conference Date: Tuesday, June 3, 2025 Time: 11:20 a.m. CT Goldman Sachs' 46 th Annual Global Healthcare Conference Date: Monday, June 9, 2025 Time: 3:20 p.m. ET A live webcast will be available on the "Events & Presentations" page in the Investors section of the Company's website at A replay of the webcasts will be archived on the Company's website for 14 days following the presentation. About CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY ® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit
Yahoo
21-05-2025
- Business
- Yahoo
CRISPR Therapeutics AG (CRSP) Strikes $95M Deal with Sirius to Develop Blood Clot Drug
CRISPR Therapeutics AG (NASDAQ:CRSP) has entered a $95 million upfront partnership with China-based Sirius Therapeutics to co-develop an siRNA drug targeting blood clotting disorders. This marks a strategic shift for CRISPR, traditionally focused on gene editing, recognizing that siRNA's reversible gene silencing offers advantages for some conditions where permanent DNA edits are risky, such as inhibiting factor XI, a clotting protein, where gene editing could cause bleeding complications. A scientist in a laboratory working on a gene editing tool, to create treatments for rare genetic diseases. Under the deal, CRISPR Therapeutics AG (NASDAQ:CRSP) will pay $25 million upfront plus a $70 million equity investment in Sirius. Both companies will share development costs and profits equally, with CRSP handling U.S. commercialization and Sirius covering Greater China. The siRNA drug, SRSD107, showed promising Phase 1 results by reducing factor XI levels up to 95% and is advancing toward a Phase 3 trial by late 2026. CRISPR Therapeutics AG (NASDAQ:CRSP)'s CEO, Samarth Kulkarni, highlighted the complementary nature of siRNA and gene editing, stating siRNA's quicker regulatory path accelerates commercialization compared to gene editing's longer timeline. The company retains excitement about its CRISPR-based cardiovascular programs, including ANGPTL3 and lipoprotein(a) gene edits, but is open to licensing more siRNA assets from Sirius. This diversified approach aims to build a best-in-class cardiovascular franchise by applying the most appropriate technology for each target. Shortly after, Piper Sandler analyst Raghuram Selvaraju maintained an Overweight rating on CRISPR Therapeutics AG (NASDAQ:CRSP) with a $50 price target, citing the Sirius collaboration as a potential near-term revenue catalyst. While we acknowledge the potential of CRSP to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than CRSP and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. Sign in to access your portfolio
Scientific American
16-05-2025
- Health
- Scientific American
In World First, Baby Receives Personalized CRISPR Gene-Editing Treatment
A baby boy with a devastating genetic disease is thriving after becoming the first known person to receive a bespoke, CRISPR therapy -for-one, designed to correct his specific disease-causing mutation. Little KJ Muldoon, now nearly ten months old, is doing well after receiving three doses of a gene-editing treatment to mend a mutation that impaired his body's ability to process protein, his parents told reporters this week. But it is too soon to use the word 'cure', says Rebecca Ahrens-Nicklas, a pediatrician at Children's Hospital of Philadelphia in Pennsylvania, and one of Muldoon's physicians. 'This is still really early days,' she says. 'We know we have more to learn from him.' To reach this point, an international team of clinicians and researchers in industry and academia, with support from US government funders and regulatory agencies, raced to develop Muldoon's therapy in a mere six months. Yet, the drug that it developed, described in the New England Journal of Medicine on May 15, is specific to Muldoon's genetic sequence and will probably never be used for another person, says Ahrens-Nicklas. On supporting science journalism If you're enjoying this article, consider supporting our award-winning journalism by subscribing. By purchasing a subscription you are helping to ensure the future of impactful stories about the discoveries and ideas shaping our world today. It's an ambitious approach that researchers hope will inspire others to harness CRISPR to treat ultra-rare genetic diseases. 'This truly is the future for all of these gene and cell therapies,' says Arkasubhra Ghosh, who studies gene therapy at Narayana Nethralaya Eye Hospital in Bengaluru, India, and who was not involved in the study. 'It's really exciting.' Early illness Dozens of people have received CRISPR-based therapies for genetic conditions such as sickle-cell anaemia, but those treatments were designed to be used in many people with the same disorder, regardless of the underlying mutations that caused it. By contrast, researchers tailored Muldoon's therapy to correct a specific genetic sequence in his genome. Muldoon had inherited two mutations, one from each parent, that meant that he did not produce the normal form of a crucial enzyme called carbamoyl phosphate synthetase 1 (CPS-1). This compromised his ability to process the nitrogen-containing compounds produced when the body breaks down protein. As a result, his blood had high levels of ammonia, a compound that is particularly toxic to the brain. The best treatment for CPS-1 deficiency is a liver transplant, but it would be months before Muldoon became eligible. Meanwhile, each day brought added risk of brain damage or death: only about half of babies with severe CPS-1 deficiency survive long enough to receive a transplant. Ahrens-Nicklas decided to offer the family another option. She and her colleagues had been working with a CRISPR-based technique called base editing, which can make targeted, single-letter changes to DNA sequences. The team was developing ways to quickly and safely tailor a base-editing therapy to correct an individual's particular mutations. Perhaps now it was time to try the approach in humans, she thought. With the approval of Muldoon's parents, the researchers enlisted a lengthy roster of collaborators. The team quickly screened for the best base-editing approach and tested it in mice and monkeys. Companies donated proprietary expertise and components. The US Food and Drug Administration fast-tracked its evaluation of the treatment. Rapid deployment In just six months, Muldoon received his first dose — a 'remarkable' achievement, says Waseem Qasim, a pediatrician at the University College London Great Ormond Street Institute of Child Health, who has used base editing to engineer immune cells to fight cancer. After that initial dose, Muldoon could safely eat the amount of protein recommended for his age, but still needed medications to keep his ammonia levels in check. With a second round of the therapy, the researchers were able to reduce the amount of medicines needed, but could not eliminate his need to take them. Muldoon has since received a third and final dose. His clinicians are carefully reducing his medication dosage, little by little, says Ahrens-Niklas. It's unclear how this approach could be expanded to treat others with ultra-rare diseases: even when designed to treat hundreds of people, gene therapies and gene-editing therapies are notoriously expensive. 'There's no great answer to this,' says Qasim. For now, each milestone that Muldoon reaches is a tiny miracle to his parents. Earlier this week, his mother, Nicole, walked into his hospital room to find him sitting up by himself in his crib. 'We never thought this was going to happen,' she says.
India Today
16-05-2025
- Health
- India Today
In world's first, doctors customise DNA to treat baby with rare liver disorder
Doctors have successfully used a customised form of gene editing to ease the symptoms of a rare and life-threatening genetic liver disorder in a baby is the first time this type of CRISPR-based technology has been used in a living human with a specific baby, identified as "KJ" by his family, was just 7 months old when he received the experimental treatment in February He was born with a severe condition called carbamoyl phosphate synthetase 1 (CPS1) deficiency, a disorder so rare it affects only one in a million disease is caused by a faulty gene in the liver, leading to dangerous build-ups of ammonia in the blood, which can cause brain damage, coma, or even death. if not managed boy's case was reported in The New England Journal of Medicine (NEJM) and at a gene therapy GENE EDITINGKJ was treated using base editing, a more precise and safer version of the better-known CRISPR gene editing standard CRISPR, which cuts both strands of DNA, base editing changes just a single letter in the DNA sequence, which minimises the risk of any and doctors from the University of Pennsylvania and Children's Hospital of Philadelphia (CHOP) custom-designed the base editor specifically to fix the mutation in KJ's CPS1 this, they developed the entire treatment, from concept to delivery, in just six editing tool was delivered through tiny fat particles called lipid nanoparticles, which were injected into KJ's bloodstream so they could reach his liver AFTER THREE DOSESThough KJ still requires a special diet and medications to help control his condition, early signs suggest gene editing is receiving three doses of the base editor, he can now tolerate more protein in his diet and needs less medicines to control his ammonia more promising, KJ recently recovered from two viral infections without the usual dangerous spikes in ammonia that typically follow in such to the report, doctors did not perform a liver biopsy to confirm the gene correction directly, as it was considered too risky for the his improved condition is "strong indirect evidence" that the treatment has worked at least in father, Kyle Muldoon, said during a press conference, "We're very, very happy with the results."KJ is expected to go home soon, doctors A CURE, BUT A MAJOR STEP FORWARDWhile KJ isn't cured, he may need additional doses in the his cause proves that personalised gene editing for rare genetic diseases is not only possible but can be done in a matter of Kiran Musunuru of Penn Medicine, one of the lead researchers of the case, said, 'Our hope is that this will be the start of something that many others around the world will pick up on.'TAILOR-MADE GENOMEThe success of this personalised base editor adds to a growing list of gene therapy approaches for rare methods include using CRISPR-like tools to insert entire healthy genes into the genome. One such treatment recently helped another baby with a similar urea cycle disorder, allowing that child to stop medication and eat a normal these treatments are still experimental and can carry risks, such as unwanted immune responses or unintended gene edits, the scientists believe they represent the future of medicine for patients with rare and deadly genetic conditions.'This seems to be safe. And there are early signs that it's going to benefit him,' said Dr. Rebecca Ahrens-Nicklas, who helped treat historic step in gene editing could soon change how doctors treat, not just manage, genetic diseases, one patient at a time. advertisement
The Star
15-05-2025
- Health
- The Star
U.S. infant becomes first to receive personalized gene therapy for rare incurable disease
LOS ANGELES, May 15 (Xinhua) -- In a groundbreaking medical milestone, U.S. researchers have successfully developed and delivered a personalized gene-editing therapy to treat an infant suffering from a rare and life-threatening genetic disorder. The child, diagnosed shortly after birth with carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the first human to receive a customized CRISPR-based treatment targeting the root cause of the condition, according to a release of the U.S. National Institutes of Health (NIH) on Thursday. CPS1 deficiency is characterized by an inability to fully break down byproducts from protein metabolism in the liver, causing ammonia to build up to toxic levels in the body. It can cause severe damage to the brain and liver, according to NIH. A team of researchers at the Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania developed the customized therapy using the gene-editing platform CRISPR. They corrected a specific gene mutation in the baby's liver cells that led to the disorder. CRISPR is an advanced gene editing technology that enables precise changes to DNA inside living cells. This is the first known case of a personalized CRISPR-based medicine administered to a single patient and was carefully designed to target non-reproductive cells so changes would only affect the patient, according to NIH. The child initially received a very low dose of the therapy at six months of age, then a higher dose later. The research team saw signs that the therapy was effective almost from the start, according to NIH. Their findings were published Thursday in The New England Journal of Medicine. "As a platform, gene editing -- built on reusable components and rapid customization -- promises a new era of precision medicine for hundreds of rare diseases, bringing life-changing therapies to patients when timing matters most: Early, fast, and tailored to the individual," said Joni L. Rutter, director of NIH's National Center for Advancing Translational Sciences.
