Latest news with #BeamTherapeutics
Business Insider
3 days ago
- Business
- Business Insider
Beam Therapeutics treatment of sickle cell disease granted orphan designation
The FDA granted orphan designation to a treatment of sickle cell disease being developed by Beam Therapeutics (BEAM), according to a post to the agency's website. Confident Investing Starts Here: Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
Business Insider
5 days ago
- Business
- Business Insider
Beam Therapeutics treatment of alpha-1 antitrypsin deficiency gets orphan status
Beam Therapeutics (BEAM)' treatment of alpha-1 antitrypsin deficiency was granted FDA orphan designation status, according to a post to the agency's website. Confident Investing Starts Here: Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
Yahoo
6 days ago
- Business
- Yahoo
Rocket Pharmaceuticals, Inc. (RCKT) Gene Therapy Trial Paused Following Patient Death
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) has suspended dosing in its Danon disease gene therapy trial after a patient developed fatal complications from capillary leak syndrome, which caused a severe drop in blood pressure and led to an acute systemic infection. The FDA placed a formal clinical hold on the trial following the incident. The company is investigating the cause, focusing on a newly introduced immune suppression agent designed to prevent complement system activation before administering the gene therapy. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is collaborating with the FDA, an independent data board, and trial investigators to review safety measures and resume the study as soon as possible. A microbiologist studying a petri dish culture of a gene therapy on a microscope. The patient's death has raised concerns among analysts about the unpredictable risks of gene therapies, with Jefferies warning that such events complicate investment decisions in the sector. However, BMO Capital Markets noted that the benefit-risk profile for RCKT's RP-A501 remains justified, given the high mortality rate in Danon disease patients. This incident adds to a series of recent safety setbacks in the gene therapy field, including patient deaths in trials by Sarepta Therapeutics, Neurogene, and Beam Therapeutics. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) reported $318.2 million in cash, providing financial runway into 2027, but analysts caution that cash burn will need close monitoring. While we acknowledge the potential of RCKT to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than RCKT and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
6 days ago
- Business
- Yahoo
Rocket Pharmaceuticals, Inc. (RCKT) Gene Therapy Trial Paused Following Patient Death
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) has suspended dosing in its Danon disease gene therapy trial after a patient developed fatal complications from capillary leak syndrome, which caused a severe drop in blood pressure and led to an acute systemic infection. The FDA placed a formal clinical hold on the trial following the incident. The company is investigating the cause, focusing on a newly introduced immune suppression agent designed to prevent complement system activation before administering the gene therapy. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is collaborating with the FDA, an independent data board, and trial investigators to review safety measures and resume the study as soon as possible. A microbiologist studying a petri dish culture of a gene therapy on a microscope. The patient's death has raised concerns among analysts about the unpredictable risks of gene therapies, with Jefferies warning that such events complicate investment decisions in the sector. However, BMO Capital Markets noted that the benefit-risk profile for RCKT's RP-A501 remains justified, given the high mortality rate in Danon disease patients. This incident adds to a series of recent safety setbacks in the gene therapy field, including patient deaths in trials by Sarepta Therapeutics, Neurogene, and Beam Therapeutics. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) reported $318.2 million in cash, providing financial runway into 2027, but analysts caution that cash burn will need close monitoring. While we acknowledge the potential of RCKT to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than RCKT and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Insider
25-05-2025
- Business
- Business Insider
Analysts Have Conflicting Sentiments on These Healthcare Companies: Novo Nordisk (NVO), Beam Therapeutics (BEAM) and TScan Therapeutics (TCRX)
Companies in the Healthcare sector have received a lot of coverage today as analysts weigh in on Novo Nordisk (NVO – Research Report), Beam Therapeutics (BEAM – Research Report) and TScan Therapeutics (TCRX – Research Report). Confident Investing Starts Here: Novo Nordisk (NVO) In a report issued on May 6, Evan Seigerman from BMO Capital maintained a Hold rating on Novo Nordisk, with a price target of $64.00. The company's shares closed last Friday at $67.35. According to Seigerman is a 4-star analyst with an average return of 3.2% and a 42.7% success rate. Seigerman covers the Healthcare sector, focusing on stocks such as Structure Therapeutics, Inc. Sponsored ADR, Protagonist Therapeutics, and Arvinas Holding Company. The word on The Street in general, suggests a Hold analyst consensus rating for Novo Nordisk with a $92.44 average price target. BMO Capital analyst Kostas Biliouris maintained a Buy rating on Beam Therapeutics on May 7 and set a price target of $40.00. The company's shares closed last Friday at $16.83. According to Biliouris has currently 0 stars on a ranking scale of 0-5 stars, with an average return of -17.1% and a 29.2% success rate. Biliouris covers the Healthcare sector, focusing on stocks such as Centessa Pharmaceuticals, BioMarin Pharmaceutical, and Rocket Pharmaceuticals. Beam Therapeutics has an analyst consensus of Strong Buy, with a price target consensus of $44.13, implying a 164.3% upside from current levels. In a report issued on May 6, William Blair also maintained a Buy rating on the stock. TScan Therapeutics (TCRX) BTIG analyst Jeet Mukherjee maintained a Buy rating on TScan Therapeutics on May 21 and set a price target of $12.00. The company's shares closed last Friday at $1.38, close to its 52-week low of $1.34. According to Mukherjee has 0 stars on 0-5 stars ranking scale with an average return of -15.8% and a 31.6% success rate. Mukherjee covers the Healthcare sector, focusing on stocks such as Arvinas Holding Company, Janux Therapeutics Inc, and Carisma Therapeutics. Currently, the analyst consensus on TScan Therapeutics is a Strong Buy with an average price target of $9.33, representing a 557.0% upside. In a report issued on May 6, TD Cowen also maintained a Buy rating on the stock.
