In a recent press release, gene therapy company Orchard Therapeutics ("Orchard") announced that the FDA approved Orchard's Investigational New Drug (IND) application for OTL-200. This gene therapy solution is…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
While gene therapy offers the promise of addressing and treating many genetic disorders, creating a better quality of life for many people, there are also ethical issues associated with…
In mid-November, 2020, Rocket Pharmaceuticals announced that the company received a $3.7M grant from the California Institute for Regenerative Medicine to study potential treatments for malignant infantile osteopetrosis. In…
Charcot-Marie-Tooth (CMT) disease is a neurological disorder with a number of subtypes. Because of this, no treatment is universally successful within all affected individuals. In order to get patients the…
Across the world, various countries have Orphan Drug or Orphan Medicinal Product designations that leading agencies grant to products designed to treat patients with rare diseases. Recently, AVROBIO announced that…
In late October 2020, Homology Medicines ("Homology") announced its development of a new gene therapy program to treat patients with MPS II, or Hunter syndrome. Homology's mission is to meet…
A new treatment for Krabbe disease, PBKR03, has recently been granted the Orphan Drug and Rare Pediatric Disease designations by the FDA, according to an article in GlobeNewswire. These designations…
Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
Currently, most treatments for scleroderma, a group of conditions causing the hardening of skin and connective tissue, are symptomatic. That is, they treat symptoms but not the underlying cause of…
According to a story from GlobeNewswire, the gene therapy company Axovant Gene Therapies Ltd. recently announced that its gene therapy AXO-AAV-GM2 has been given Rare Pediatric Disease designation by the…
In recent years, there have been huge progressions in the field of gene therapy. At its core, gene therapy addresses genetic disorders by using genes to directly treat or…
The FDA has recently granted two designations, the rare pediatric disease and orphan drug designations, to Spirovant Sciences' gene therapy for cystic fibrosis (CF). SPIRO-2101 will now be able to…
On October 2, 2020, biotechnology company BioMarin Pharmaceutical ("BioMarin") announced that its investigational gene therapy candidate BMN 307 received the Fast Track designation from the FDA. The therapy is designed…
Seelos Therapeutics and Duke University have both signed a Sponsored Research Agreement, meaning they will work in tandem to evaluate and test SLS-004, a Parkinson's disease treatment. Their research aims…
According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet's gene therapy, which treats Wilson disease. The terms of the agreement…
BioMarin has been working to develop treatments for phenylketonuria for 15 years, and it is a cause they are very committed to. That is why they are so excited to…
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
Several decades ago, gene therapy seemed to be on target to treat a disease by simply replacing a defective gene with a healthy one. A recent article republished in APNews…
According to an article from ScienceBlog, a twist on CRISPR gene editing has allowed a team of researchers to successfully treat myotonic dystrophy type 1 in mouse models. By modifying…
Last month, biotechnology company BioMarin sought approval for an investigational gene therapy (valoctocogene roxaparvovec) for patients with hemophilia A. However, their approval was denied. Instead, the company was told that…
Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
In recent years, gene therapy has expanded to enormous heights. This experimental technique allows scientists to add, delete, edit, or otherwise use genes to address genetic malfunctions and disorders. Now,…
According to Charcot-Marie-Tooth News, biotechnology company Helixmith recently began a Phase 1/2a clinical trial to test the efficacy of Engensis (VM202) for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). The…
Last Tuesday, the FDA rejected BioMarin Pharmaceutical's gene therapy, Roctavian, for patients with hemophilia A. According to ABC News, the gene therapy infusion was meant to be a single treatment…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
