Sanofi dominates the market for therapies that treat Pompe disease, a rare enzyme deficiency. The FDA has approved an Amicus Therapeutics combination treatment for patients inadequately served by the Sanofi products.
Dr. Ala Stanford, founder of the Black Doctors Covid-19 Consortium, provided hundreds of thousands of Covid-19 tests and vaccines to Black and Brown people in Philadelphia's underserved neighborhoods during the pandemic. In her view, her work was impactful because she acted promptly and focused on community-based patient engagement.
As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers.
VintaBio manufactures AAV and lentiviral vectors for entities pursuing clinical development of cell and gene therapies. The contract development manufacturing organization’s launch comes as demand for viral vectors continues to outpace the supply of these crucial components of the therapies.
Capstan Therapeutics, a University of Pennsylvania spinout, is now out of stealth with technology that could enable new medicines that work by engineering cells inside of a patient. Penn biotech pioneers Carl June and Drew Weissman are among the scientific co-founders of this startup, which aims to develop therapies for fibrosis, cancer, inflammation, and genetic diseases.
The SPAC merger that Amicus Therapeutics planned for its gene therapy assets has been called off due to market conditions. The rare disease company is instead keeping those assets but streamlining its portfolio in order to save $400 million—the amount that the gene therapy spinout was in line to receive from the merger deal.
Bristol Myers Squibb is partnering with Century Therapeutics, placing a $150 million bet that the biotech’s technology for engineering stem cells can produce new off-the-shelf cell therapies for cancer. If the four potential programs covered under the pact reach the market, milestone payments could bring Century more than $3 billion.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
Strados Labs has developed a wearable device that senses lung sounds in patients with asthma, COPD, and other respiratory disorders. The startup, which has FDA clearance and a growing list of customers, was judged the winner in the Pitch Perfect competition at MedCity News’ recent INVEST Digital Health conference.
The startup shared results of a prospective study showing its deep learning software detected melanoma with 93% sensitivity and 91% specificity.
Amicus Therapeutics is spinning its gene therapy pipeline into a separate entity that will go public via a SPAC merger. Executives say the transaction puts Amicus on a path to profitability while simultaneously securing the cash needed to develop the gene therapy assets.
Biotech startup iECURE is based on work from the University of Pennsylvania scientist, who has been researching ways to use in vivo gene editing as a way to “knock in” healthy versions of a gene to treat rare liver diseases. The company, which will develop therapeutic candidates from Penn, has raised $50 million in Series A financing.
The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.
With the extension to its Series A financing, XyloCor plans a clinical trial testing its gene therapy as an adjunct to coronary artery bypass graft surgery. A study in refractory angina is already underway.
Proscia and other digital-pathology firms promise to make pathologists more efficient as they analyze tissue. The need is driven by a steadily declining U.S. population of pathologists.
A review published in JAMA found that bills introduced into the House and Senate don't go nearly as far as existing drug pricing regulations other countries. The researchers listed eight key lessons from six nations.
The charges, filed in Philadelphia federal court, allege that Teva conspired with four other generic drug companies to fix prices, rig bids and allocate customers. Teva - which the Justice Department and New York's state government also sued this month for unrelated allegations - said it rejected the allegations.
One study investigator pointed to the data for ADP-A2M4 as indicating much greater efficacy in the rare cancer than existing therapy options, while another said it demonstrated cell therapies' potential outside of the blood cancer space.