Latest news with #geneTherapy
Forbes
a day ago
- Health
- Forbes
Gene Therapy For Inherited Disease In The Unborn Child
Until recently, even the most advanced gene therapies could only be given after a child was ... More born—often racing against time to prevent irreversible damage. In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns and their families. Now, we're taking an even earlier step: treating inherited diseases in the womb before birth. Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. But what if we could intervene even earlier? That's the question now being answered, with some astonishing results. A recent study published in Science Translational Medicine showed that delivering a special kind of genetic therapy directly to the fetus could prevent the onset of spinal muscular atrophy in animal models. Treating the condition before birth may be possible to preserve healthy motor function and prevent the nerve damage that usually begins in the womb. This is the first time we see molecular therapies targeting the root cause of inherited disease before birth. Another special investigational case, the first in humans, found that providing the mother with gene therapy while pregnant and continuing treatment after birth also prevented the devastating muscle weakness that usually comes with the disease. This is a true leap forward: instead of managing symptoms; we may soon be able to stop some inherited diseases before they ever begin. The journey starts with advanced prenatal genetic screening. Genetic changes in the developing fetus can be found using a simple blood sample from the mother. When a risk is found, therapy is delivered directly to the fetus, often by injecting medicine into the amniotic fluid. In the case of spinal muscular atrophy, this approach in animal models led to healthier development and longer survival. These findings suggest that intervening before birth can prevent or significantly reduce the neurological damage that begins in the womb and progresses rapidly after birth. While most of this research has been in animals, the first human steps have already begun. In February 2025, the University of California, San Francisco, reported the world's first attempt to treat a genetic disease in a human fetus using a medication called risdiplam. After learning that her unborn child was at risk, a mother began taking the medication late in pregnancy. The baby was born healthy and—now more than two years old—shows no signs of the disease, though some developmental challenges remain. Another important step was taken in a clinical trial at UCSF, where doctors successfully treated a fetus with a different rare disease using enzyme replacement therapy, showing that the technology for delivering medicine to the unborn is already here. Many inherited diseases cause the most significant harm before a baby is even born. By intervening early, we have the chance to save lives and give children the best possible start—preserving their ability to move, think, and grow. This isn't just a medical advance. It's a new way of thinking about what's possible for families facing genetic disease. Of course, there are still challenges ahead. Many are working to ensure these therapies are safe, effective, and accessible to all who need them. Ethical questions about when and how to use these powerful tools will also need careful thought. The first human applications of gene therapy before birth are expected within the next decade, pending rigorous safety and ethical evaluations. This new era also brings new questions. If we correct a genetic error in a child before birth, will that change be passed on to future generations? For now, most therapies target the body's somatic cells, not the germline, so the changes are not inherited. However, the line between somatic and germline interventions may blur as technology evolves, raising complex ethical considerations. The first human trials of in-utero gene therapy are just beginning, and more research is needed. But the direction is clear: as technology advances, we are moving from treating inherited diseases after birth to preventing them at the start of life. As I have often said, the future of medicine is being rewritten, one gene at a time. This latest breakthrough brings us one step closer to a world where prevention, rather than treatment, becomes the standard for genetic disease, where every child can live their healthiest life from the very start. As discussed in my book, the hope is that every child will soon have the chance to live their healthiest life from the beginning.
Associated Press
2 days ago
- Business
- Associated Press
Abeona Therapeutics® Announces New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
CLEVELAND, June 02, 2025 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced it has granted equity awards to new non-executive employees who joined the Company. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4). On May 31, 2025, the Compensation Committee of Abeona's Board of Directors granted restricted stock equity awards as a material inducement to employment to five individuals hired by Abeona, which equity awards relate to, in the aggregate, up to 11,500 restricted shares of Abeona common stock. One-third of the shares subject to such restricted stock awards will vest yearly on each anniversary of the Grant Date, such that the shares subject to such restricted stock awards granted to each employee will be fully vested on the third anniversary of the Grant Date, in each case, subject to each employee's continued employment with Abeona on the applicable vesting dates. About Abeona Therapeutics Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona's ZEVASKYN™ (prademagene zamikeracel) is the first and only autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The Company's fully integrated cell and gene therapy cGMP manufacturing facility in Cleveland, Ohio serves as the manufacturing site for ZEVASKYN commercial production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit ZEVASKYNTM, Abeona AssistTM, Abeona Therapeutics®, and their related logos are trademarks of Abeona Therapeutics Inc. Forward-Looking Statements Investor and Media Contact: Greg Gin VP, Investor Relations and Corporate Communications Abeona Therapeutics [email protected]
Yahoo
5 days ago
- Business
- Yahoo
bluebird bio, Inc. (BLUE) Nears Acquisition by Carlyle and SK Capital
Investment firms The Carlyle Group Inc. (NASDAQ:CG) and SK Capital Partners, together with Beacon Parent Holdings, L.P., have successfully completed the tender offer for bluebird bio, Inc. (NASDAQ:BLUE) to acquire all outstanding common stock. With a value of either $3.00 per share plus a contingent value right or $5.00 per share in cash, the transaction is set to be finalized through a merger on June 2, 2025. A medical researcher inspecting a petri-dish filled with bacteria in a laboratory setting. We have previously seen how bluebird bio, Inc. (NASDAQ:BLUE), a biotechnology company engaging in gene therapies for serious genetic diseases, was both out of time and out of money. Among the challenges faced by the company were financial burdens, a high debt-to-capital ratio of 0.82, and a low current ratio of 0.55. In February, the management reported a deal with Carlyle and SK Capital Partners for $3 in cash and a $6.84 contingent value. Soon after, the entities secured approvals from the EU, China, and Turkey, meeting all the regulatory requirements. Under the terms of the agreement, the transaction is anticipated to advance without a vote from the bluebird bio, Inc. (NASDAQ:BLUE) stockholders, complying with Delaware's General Corporation Law. By offering a choice to the shareholders through an amended buyout agreement, the company provides immediate cash to those who desire it instead of potential future payments. READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure. None. Sign in to access your portfolio
Washington Post
28-05-2025
- Health
- Washington Post
Scientists develop gene delivery ‘trucks' that could treat brain diseases
Scores of researchers have produced new tools that can deliver genes and selectively activate them in hundreds of different cell types in the brain and spinal cord, a breakthrough that scientists hope advances them toward developing targeted therapies to treat neurodegenerative diseases such as ALS, Parkinson's disease and Alzheimer's. The discoveries, made through the National Institutes of Health's BRAIN initiative, show with unprecedented clarity and precision how neural cells work together, but also how diseases disrupt their tight choreography. The insight offers the promise that doctors may one day treat diseases by manipulating dysfunctional cells.
Reuters
27-05-2025
- Business
- Reuters
FDA halts Rocket Pharma's gene therapy trial as complications lead to patient death
May 27 (Reuters) - The U.S. FDA has paused a mid-stage gene therapy trial by Rocket Pharmaceuticals (RCKT.O), opens new tab after a patient suffered serious complications, which ultimately led to the person's death, the drug developer said on Tuesday. Shares of the New Jersey-based company tumbled 63% to $2.30 in premarket trading. They had closed at $6.27 on Friday. The company said the FDA had placed a hold on the trial on Friday after the report of a capillary leak syndrome, a condition where fluid and proteins leak out of tiny blood vessels into surrounding tissues, causing swelling and low blood pressure. The patient had subsequently died, it said. Rocket was testing its experimental gene therapy, RP-A501, for the treatment of Danon disease, a rare genetic disorder that causes heart muscle damage and progressive muscle weakness. The company said it had voluntarily paused further dosing in the study. It is also working with the U.S. Food and Drug Administration and experts to resolve the trial halt and resume the study quickly, but cannot provide a timeline for completion until the clinical hold is lifted. The FDA did not immediately respond to a Reuters request for comment.
