Protalix Biotherapeutics Inc. and the Chiesi Group’s global rare diseases unit gained approval of Elfabrio (pegunigalsidase alfa-iwxj) from the U.S. FDA for adults with Fabry disease. The decision comes shortly after the go-ahead was granted for the drug in Europe. A modified stabilized version of the recombinant human alpha-galactosidase A protein, the enzyme replacement therapy (ERT) Elfabrio was previously known as PRX-102. Shares of Protalix (NYSE:PRX) were trading midday at $2.88, down 4 cents.

Sobi entering myelofibrosis fray with $1.7B offer for CTI Biopharma

Swedish Orphan Biovitrum (Sobi) AB is offering a hefty 95% premium to shareholders in CTI Biopharma Corp., as it tables a $9.10-per-share cash offer for the company, which implies a total equity value of $1.7 billion. Shares in Seattle-based CTI (NASDAQ:CTIC) had closed May 9 at $4.82 but surged almost 87% to a high of $8.99 during premarket trading May 10 once the deal was disclosed. In contrast, shares in Solna, Sweden-based Sobi (Stockholm:SOBI) fell by as much as 17% to SEK216 (US$21.12) during early trading. The transaction will involve some dilution for shareholders. Sobi will finance it by debt initially, but it plans to recoup about half of the total outlay through a subsequent rights issue.

Adcom briefing docs less than upbeat about Sarepta’s DMD therapy

Briefing documents related to the closely watched regulatory meeting to deliberate Sarepta Therapeutics Inc.’s gene transfer therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD) were made public. The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee will meet May 12 to discuss the compound, also known as SRP-9001. In the briefing docs, FDA reviewers worry, among other things, about microdystrophin as a surrogate for function in DMD. Shares of Sarepta (NASDAQ:SRPT) were trading midday at $129.74, up $4.37.

Bayer on board, too: Bicycle inks second $1.7B radioconjugate deal in as many months

Barely a month and a half after its radioconjugate approach landed a potential $1.7 billion deal with Novartis AG, Bicycle Therapeutics plc drew another big radiopharma player to the table, signing a collaboration agreement with Bayer AG to use Bicycle’s peptide technology to discover and develop radiotherapies against cancer targets. Terms are similar to the Novartis deal, with Bayer paying $45 million up front and Bicycle eligible for development and commercial milestones totaling up to $1.7 billion, plus royalties, and underline the growing interest in the radiopharma space.

Bye bye, bardo: Reata halts development in kidney disease

Despite hitting a phase III study’s primary and key secondary endpoints for treating diabetic kidney disease, Reata Pharmaceuticals Inc. is discontinuing its programs for the indication that use bardoxolone. The data that disappointed Reata were, after three years of treatment, finding no separation in end-stage renal disease events between the bardoxolone and placebo groups. Because of the halt, the company’s development and funding agreement with Blackstone Life Sciences has been amended to remove all of bardoxolone’s development and commercial cost obligations, a savings of about $100 million. Reata and Kyowa Kirin Co. Ltd. have been developing the treatment since 2009. Reata’s stock (NASDAQ:RETA) had buckled at midday, with shares trading 22% lower at about $82.95 each.

Diogenx closes $30M series A round to boost beta-cell replication in diabetes

Diogenx SAS raised €27.5 million (US$30.4 million) in a series A round to move a novel therapy for type 1 diabetes (T1D) into clinical development. The Marseille, France-based company is building on the research of co-founder Patrick Collombat, an expert in beta-cell regeneration, who is based at the Insitute of Biology Valrose and the University Côte d’Azur, in Nice. Its lead drug candidate comprises a recombinant R-spondin protein, which acts on the Wnt/beta-catenin signal pathway to boost replication of endogenous functioning beta-cells.

BIO Korea 2023: South Korea takes large strides in gene therapy, regenerative medicine

When it comes to CAR T therapies, South Korea is trailing behind the U.S. and China, but the South Korean government sees cell and gene therapies as a space where the country can draw international investors, speakers said during the Bio Korea 2023 conference in Seoul May 10 to 12. Icm Co Ltd. is developing recombinant adeno-associated virus (rAAV) gene therapy for a number of degenerative diseases. “Korea is leading drug development in AAV gene therapy, and most countries are in early clinical trials,” said CEO Dae-Won Kim. ICM, along with other Korean companies, are leading the way with AAV therapies in osteoarthritis, rheumatoid arthritis, and diabetic retinopathy, he said.

Pangenome gives more panoramic view of human diversity

The human genome, the sequence that represents the DNA of our species, was built with a single individual as a model. This all-in-one standard didn’t include the gene variations that make us different or explain why some people develop certain diseases. Four simultaneous studies from the Human Pangenome Reference Consortium have published a sequence based on 47 individuals, beginning to capture the genetic diversity that defines humans. The pangenome starts here and it will go on expanding the knowledge of genetics. From a medical point of view, a greater diversity of the known genome means delving into the genetic basis of disease, its diagnosis and better treatments.

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