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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.

First Patient Dosed in Phase 3 Trial of Etrasimod for Moderate to Severe UC

The Phase 3 trial ELEVATE UC 52 evaluating Arena Pharmaceuticals’ investigational therapy etrasimod for patients with moderately to severely active ulcerative colitis has dosed its first patient. “We are pleased to enroll the first patient in the ELEVATE UC trial, supporting etrasimod’s potential as an important future therapy for ulcerative colitis,” Darshan Anandu, MD, of G.I. Specialists of Houston, said in a press release.

With $45M Nurix Deal, Gilead Enters Crowded Protein Degradation Field

Gilead Sciences is turning to Nurix Therapeutics in an effort to discover new drugs that harness the cellular machinery our bodies use to dispose of damaged or harmful proteins.

SanFranBusTimes

Why Gilead could pay more than $2 billion in deal with this small S.F. biotech

Founded by three professors from UC Berkeley and UCSF, Nurix’s protein-degradation approach to cancer, and potentially other diseases, could stop cancer in its tracks.

The Differences Between Device and Drug Studies

Device and drug trials pose a variety of challenges; they are, after all, completely different in study design, have varying regulatory pathways, as well as differing executional challenges. In this interview, Tony Fiorino, Chief Medical Officer of electroCore, will discuss challenges he has faced with device studies, and will elaborate on his experiences about the differences between device and drug trials.

Arqule sees strength in early BTK inhibitor data; shares rise on phase I CLL news

Shares of Arqule Inc. (NASDAQ:ARQL) hit a 52-week high Friday on news that the company’s Bruton’s tyrosine kinase (BTK) inhibitor, ARQ-531, achieved partial responses in four of six heavily pretreated people with relapsed/refractory (r/r) chronic lymphocytic leukemia (CLL) during an ongoing phase I trial.

Terumo plans to take Orchestra BioMed’s sirolimus balloon international in $35M deal

Orchestra BioMed is partnering up with Terumo to help develop and commercialize its sirolimus-eluting balloon in both coronary and peripheral cardiovascular interventions. The Virtue angioplasty system is designed to deliver a bioabsorbable, sustained-release formulation of sirolimus directly into the artery wall. The drug helps prevent the vessel from narrowing and reclogging following the placement of a stent or other intervention, a process known as restenosis.