ProQR: RNA Therapy Has Potential to Restore Vision in Patients With Usher Syndrome
Usher syndrome is an inherited retinal degeneration that affects hearing, balance, and vision. Usher syndrome caused by USH2A mutations is one of the most common causes of retinitis pigmentosa (RP) with syndromic features. Currently there is no treatment for the sensory deficits caused by Usher syndrome.
In an attempt to fill this void, QR-421a is an antisense oligonucleotide (ASO) and designed to specifically target mutations in exon 13 of the USH2A gene for the treatment of patients with RP.
Orchestra Biomed raises $34M to advance circulatory solutions
New Hope, Pa.-based Orchestra Biomed Inc. scooped up $34 million in a series B-1 preferred stock financing led by Perceive Advisors, RTW Investments and Soleus Capital. The funds will be used to advance development of the company’s Backbeat cardiac neuromodulation therapy (CNT) system, to support commitments to Orchestra’s strategic global partnership with Tokyo-based Terumo Corp. for the development and marketing of the Virtue sirolimus-eluting balloon (SEB) and to grow the company’s product pipeline and pursue future collaborations.
The Cell Therapy Discovery About To Disrupt Clinical Trials
Armon Sharei developed an interest in novel cell therapies while pursuing his PhD in chemical engineering at MIT. While pursuing that interest, he made a discovery that today is the foundation of SQZ Biotech, a cell-therapy company where he serves as CEO. Sharei discovered a new method of inserting materials into cells more effectively than anything that currently existed. His discovery is now opening doors to what researchers can force cells to do, and, as a result, what cells can do for patients.
My sore throat turned out to be a brain tumor: Girl, 19, describes shock diagnosis that required innovative new surgical technique to remove the lump
For years, Christina Giuffrida had been experiencing an on-again, off-again sore throat. The 19-year-old from Yonkers, New York, also had slight balance issues and would struggle to hear people speaking at normal volumes. One doctor told her and her parents, it was a problem with her eyesight. Another said it was a flare-up from a previous mononucleosis infection or allergies.
It’s time to find new targets for brain diseases instead of just pursuing old ones
Ifirst learned about drug discovery and development in the mid-1990s when two of my sons were diagnosed with a brutal, untreatable genetic disease called ataxia-telangiectasia (A-T). Children with A‑T struggle with immune deficiency and lung problems, and have an extremely high cancer risk.
Check-Cap Goes Global with Colorectal Cancer Diagnostic Device
If you know that there’s a way to detect cancer early, would you get screened? Many people would say yes, but statistics disagree. About 40 percent of Americans do not get screened for colorectal cancer because they refuse to get or cannot get a colonoscopy. According to the American Cancer Society, 51,000 people in the US are estimated to die from colorectal cancer this year.
With phase 2 update, Krystal Biotech eyes phase 3 for rare skin disease gene therapy
Krystal Biotech unveiled data from a small phase 2 study showing its topical gene therapy closed the majority of wounds in patients with a rare skin disorder. The company is waiting on data for how long the wounds stayed closed after treatment before it moves the program into phase 3. It expects to start the pivotal trial by the end of the year.
Gene therapy biotech sees its stock rocket higher on promising results for rare cases of butterfly disease
Shares of Krystal Biotech took off this morning $KRYS after the little biotech reported promising results from its gene therapy to treat a rare skin disease called epidermolysis bullosa.
