Regenxbio Regenxbio (RGNX) announced that it is selling a portion of its royalty rights from the net sales of Zolgensma® by Novartis to entities managed by Healthcare Royalty Management, LLC (HCR) for $200 million. This transaction provides immediate, non-dilutive capital to Regenxbio in order continue the development of its gene therapy pipeline products and complete its internal manufacturing capabilities. Morgan Stanley & Co. LLC served …

Compugen Exclusive License Agreement with AstraZeneca In April 2018 Compugen (CGEN) and AstraZeneca (AZN) entered into . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Gilead Sciences Company, Kite Pharma, News Kite Pharma, a Gilead Company (GILD), announced that the European Commission has granted conditional marketing authorization for Tecartus™ (autologous, anti-CD19-transduced CD3+ cells; formerly KTE-X19). Tecartus is a chimeric antigen receptor (CAR) T cell therapy for adult patients with relapsed or refractory mantle cell lymphoma after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor. Conditional …

Intellia Therapeutics in the News Intellia Therapeutics (NTLA) presented the first demonstration of physiological protein levels of human alpha-1 antitrypsin (AAT) in non-human primates (NHPs) following a single administration.  Intellia Therapeutics Liver Gene Insertion Technology Intellia’s targeted liver gene insertion technology has the ability to achieve therapeutic levels of protein . . . This content is for paid subscribers. Please click here to subscribe or here to …

Acquisitions  AstraZeneca Acquires Alexion Pharmaceuticals AstraZeneca (AZN) and Alexion Pharmaceuticals (ALXN) entered into a definitive agreement for . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

CRISPR, Intellia and Editas, Our Gene-Editing Sisters, are Rallying The three CRISPR gene-editing firms that we have chosen are enjoying rallies. CRISPR Therapeutics (CRSP) and Intellia Therapeutics (

Reata Pharmaceuticals: Why its Stock Price is Down Reata Pharmaceuticals (RETA) announced that it has priced a public offering of 2,000,000 shares of its Class A common stock at a price to the public of $140.85 per share for gross proceeds of $281.7 million. Reata has granted the underwriters a 30-day option to purchase 300,000 additional . . . This content is for paid subscribers. …

Intellia Therapeutics Intellia Therapeutics (NTLA) stock price headed south probably because some robots learned that the firm commenced an underwritten public offering of $150 million of shares of its common stock. In addition to the commenced public offering Intellia announced its intention to grant the underwriters a 30-day option to purchase up to an additional fifteen percent (15%) of the shares . . . This content is …

Regenxbio Dosed First Patient with RGX-111 for MPS I Regenxbio (RGNX) has dosed the first patient with RGX-111 in the Phase I/II trial for the treatment of Mucopolysaccharidosis Type I (MPS I). About Regenxbio Product RGX-111 RGX-111 is an investigational one-time gene therapy designed to deliver the α-l-iduronidase (IDUA) gene directly to the central nervous system (CNS) using the NAV AAV9 vector. RGX-111 is a …

Rhythm Pharmaceuticals FDA Approval of IMCIVREE™ for Genetic Obesity At the end of the trading week last Friday, November 27, 2020, the United States FDA approved Rhythm Pharmaceuticals’ (RYTM) product IMCIVREE™ (setmelanotide). The product is designed to treat chronic obese adult patients and pediatric patients 6 years and older with obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/Kexin type . . . This content is …

Reata Pharmaceuticals We picked Reata Pharmaceuticals (RETA) for investment after confirming that the products the Company has created and is currently developing have a novel mechanism of action that enables the treatment of severe diseases that have yet to find treatments. Reata’s strategy is to build a deep understanding of the cellular response to injury in order to develop . . . This content is …

Alnylam Announces FDA Approval of OXLUMO™ Injection for PH1 Alnylam (ALNY) announced that the US FDA has approved its subcutaneous OXLUMO™ (lumasiran) injection, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. Primary Hyperoxaluria Type 1  PH1 is an ultra-rare genetic disease. It is . . . This content is for …

COVID-19 Vaccine Updates Pfizer's (PFE)/BioNTech's (BNTX) and Moderna . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Regeneron Pharmaceuticals Receives EUA from the FDA for REGEN-COV2 Regeneron Pharmaceuticals’ (REGN) antibody cocktail therapy casirivimab and imdevimab, known also REGN-COV2 or REGEN-COV2, received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA). The FDA EUA is for the treatment of mild to moderate COVID-19 in adults as well as pediatric patients at least 12 years of age and weighing at least …

Pfizer, BioNTech & Moderna When asked whether we heard the news about the submission of the Emergency Use Authorization (EUA) and whether we will inform our readers about it. Our answer was that we have posted many articles about Pfizer's (PFE) and BioNTech’s (

Gilead Sciences News Results from Phase 2/3 CAPELLA trial evaluating Gilead Sciences (GILD) lenacapavir achieved the primary endpoint in heavily treatment-experienced people with multidrug-resistant HIV-1 infection. The study showed that 88% of participants receiving lenacapavir experienced at least a 0.5 log10 reduction in HIV-1 viral load by the end of 14 days of functional monotherapy as . . . This content is for paid subscribers. Please click here …

PTC Therapeutics Approvals and Achievements PTC Therapeutics (PTCT) has achieved a lot recently. The latest is the August approval from the U.S. FDA of its product Evrysdi (risdiplam), the first orally administered at-home treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older.   Evrysdi has been granted approvals in Brazil, Chile and Ukraine as . . . This content is …

Inovio Announced FDA Clearance to Proceed with Trial for COVID-19 Vaccine Candidate Inovio (INO) announced the receipt of the U.S. FDA clearance to proceed with the Phase 2 segment of its planned Phase 2/3 clinical trial for INO-4800, its COVID-19 vaccine candidate. Inovio Planned Phase 2/3 INNOVATE Trial The INNOVATE trial - a . . . This content is for paid subscribers. Please click here to …

Moderna Has Successful COVID-19 Vaccine Results and News Moderna’s Phase 3 study met statistical criteria with a vaccine efficacy of 94.5%. Moderna intends to submit for an Emergency Use Authorization (EUA) with U.S. FDA in the coming weeks and expects the EUA to be based on the final analysis of 151 cases and a median follow-up of more than 2 months. Moderna (MRNA) announced that …

Moderna Inc COVID-19 Vaccine Candidate Moderna Inc (MRNA) announced the completion of the case accrual for the first interim analysis of Phase 3 COVE study of mRNA-1273, its COVID-19 vaccine candidate. The firm announced a significant increase in the rate of case identification across sites in the last week. As a result, the Company expects the first interim analysis will include substantially more than 53 …