Recent News

• FDA Authorizes Invivyd’s Pemgarda to Prevent COVID-19 in Immunocompromised People
  BioSpace – March 25, 2024
  The FDA granted an Emergency Use Authorization to Invivyd’s monoclonal antibody Pemgarda (pemivibart) for the prevention of COVID-19 in moderately-to-severely immunocompromised individuals 12 years of age and older. Pemgarda’s Emergency Use Authorization (EUA) does not cover its use to treat COVID-19 or as post-exposure prophylaxis for people who come in contact with someone positive for the SARS-CoV-2 virus.
• First Gene Therapy for Children With Metachromatic Leukodystrophy Approved by FDA
  HealthDay – March 22, 2024
  The U.S. Food and Drug Administration has approved Lenmeldy (atidarsagene autotemcel) as the first gene therapy for the treatment of children with metachromatic leukodystrophy (MLD). Lenmeldy is a one-time, individualized single-dose infusion made from a patient’s own hematopoietic stem cells, which have been genetically modified to include functional copies of the ARSA gene that codes for the arylsulfatase A enzyme. It is indicated for presymptomatic late infantile, presymptomatic early juvenile, or early symptomatic early juvenile MLD. The priority-review orphan drug approval was based on data from 37 children who received Lenmeldy. Compared with untreated children, Lenmeldy significantly reduced the risk for severe motor impairment or death.
   
• Experimental gene therapy for giant axonal neuropathy shows promise in NIH clinical trial
  National Institutes of Health – March 20, 2024
  An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health (NIH). Currently, there is no treatment for GAN and the disease is usually fatal by 30 years of age. Fourteen children with GAN, ages 6 to 14 years, were treated with gene transfer therapy at the NIH Clinical Center and then followed for about six years to assess safety. Results of the early-stage clinical trial appear in the New England Journal of Medicine.
   
• Lab merger and expansion to spur innovation
  University of California, Riverside – March 19, 2024
  UC Riverside is advancing innovation and economic development by combining two highly specialized laboratories on campus. The initiative will expand access and capabilities for academic researchers as well as private sector users, including those working to commercialize UCR innovations. The new laboratory will be formed by merging the existing Nanofabrication Facility with the Central Facility for Advanced Microscopy and Microanalysis, or CFAMM.
• This company’s first-ever CRISPR-based drug is changing how we treat sickle cell disease
  Fast Company – March 19, 2024
  Vertex Pharmaceuticals is No. 11 on the list of the World’s 50 Most Innovative Companies of 2024. Explore the full list of companies that are reshaping industries and culture. Until late 2023, there was just one cure for sickle cell disease: a bone marrow transplant from a matched donor. The vast majority of patients for whom this wasn’t an option could only hope to use medication and blood transfusions to manage the pain and potential organ damage that results from the illness.
• Gene therapy Zolgensma called ‘medical miracle’
  WKYC – March 19, 2024
  There are few things worse than being a physician who can offer no hope to a patient or their family. But not long ago, when faced with a diagnosis of Spinal Muscular Atrophy, or SMA, parents were sent home with children who would often die before the age of two. But medical miracles and advancing science happens and now there’s a new therapy that seems to have flipped the switch on a once destined diagnosis. For the first time, Jessica Burchett of Akron and Rebecca Mulhall of Cleveland are meeting face to face, but they’ve supported each other for more than a year.
   
• Merck’s Keytruda excels in cervical cancer trial, showing an ability to extend lives. Will a broader FDA approval follow?
  Fierce Pharma – March 15, 2024
  Two months after Keytruda became the first PD-1 drug to be approved in the United States in combination with chemoradiotherapy (CRT) to treat patients with stage 3 to 4a cervical cancer, Merck’s superstar immunotherapy has strengthened its resume in the indication. A pre-specified interim analysis from the KEYNOTE-A18 trial found that the combo regimen significantly improved overall survival (OS) versus CRT alone in patients with earlier-stage cervical cancer, the company reported. In meeting its primary endpoint of OS for the treatment of newly diagnosed patients with high-risk locally advanced cervical cancer, Keytruda could be set up for approval for a broader population in the indication. It is the first immunotherapy-based regimen to demonstrate a statistically significant improvement in keeping these patients alive longer.
• Bristol Myers cell therapy wins first-of-its-kind approval
  Biopharma Dive – March 15, 2024
  For the first time, the Food and Drug Administration has approved a cell therapy for hard-to-treat forms of two blood cancers. Sold as Breyanzi, the therapy is now cleared for use in certain adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma who have received at least two prior lines of therapy. There is no established standard of care for such patients, so the FDA’s decision to grant an accelerated approval provides a much-needed and potentially transformative option, according to Breyanzi’s developer, Bristol Myers Squibb. The FDA based its decision on results from a clinical trial that evaluated just shy of 120 participants.
• UC Berkeley Launches New Molecular Therapeutics Initiative to Accelerate Drug Discovery
  Business Wire – March 14, 2024
  The University of California, Berkeley Molecular Therapeutics (MTx) division of the Molecular & Cell Biology department today announced the launch of a new initiative to accelerate drug discovery at the interface of academia and biotech. The UC Berkeley Molecular Therapeutics Initiative (MTI) will create a foundational bridge between fundamental research in rare neurological and metabolic diseases and drug discovery to identify and accelerate novel therapeutic modalities into the clinic.
• Clinical trial may offer relief for rare autoimmune disease
  UC Davis Health – March 11, 2024
  A clinical trial involving UC Davis Health suggests the investigational drug Seladelpar is a potential novel treatment for patients with the rare, autoimmune disease primary biliary cholangitis. The drug helped reduce cholestasis and liver injury, and led to significantly less itching for patients. The results of the clinical trial were published in The New England Journal of Medicine. Primary biliary cholangitis is a disease that damages the bile ducts in the liver and can lead to liver failure. It slowly degrades the ducts, which makes it harder for bile to flow from the liver, which in turn further damages the liver.
• The Cystic-Fibrosis Breakthrough That Changed Everything
  The Atlantic – March 7, 2024
  They call it the Purge. You have experienced, in a modest way, something like it in the waning days of a bad cold, when your lungs finally expel their accumulated gunk. The rattle in your chest quiets. Your sinuses clear. You smell again: the animal sweetness of your children’s hair, the metallic breeze stirring a late-summer night. Your body, which oozed and groaned under the yoke of illness, is now a perfectly humming machine. Living is easy—everything is easy. How wonderful it is to breathe, simply breathe.