10 Best Hot Stocks To Buy Right Now

6. Dyne Therapeutics, Inc. (NASDAQ:DYN)

Number of Hedge Fund Holders: 38

Year-to-Date Share Price Gains: 166.16%

Average Price Target Upside: 51.04%

Dyne Therapeutics, Inc. (NASDAQ:DYN) is a clinical-stage company dedicated to creating innovative treatments for genetically driven muscle diseases. It is focused on developing targeted oligonucleotide therapies for serious muscle diseases using its proprietary FORCE platform.

The platform is designed to improve the delivery of treatments to muscle tissue and it aims to stop or reverse disease progression. The company is currently concentrating on myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD), and facioscapulohumeral muscular dystrophy (FSHD).

The FORCE platform uses engineered antigen-binding fragments (Fabs) that target the TfR1 receptor, which is highly expressed on muscle cells. The approach offers advantages over traditional monoclonal antibodies, such as better tissue penetration, increased tolerability, and reduced immune activation risks.

It combines these Fabs with a linker that has demonstrated safety and efficacy to deliver therapeutic payloads, including antisense oligonucleotides (ASOs) and small molecules, to address the genetic causes of diseases.

The company aims to expand its portfolio by exploring additional muscle-related conditions, including rare skeletal, central nervous system, cardiac, and metabolic muscle diseases, while ensuring that its selected therapeutic payloads effectively target the genetic basis of these disorders.

On September 3, Dyne (NASDAQ:DYN) announced positive results from its Phase 1/2 DELIVER trial of DYNE-251, a treatment for Duchenne muscular dystrophy DMD targeting exon 51 skipping (skipping over faulty parts of the gene that are responsible for producing dystrophin). Dystrophin is a key protein that helps maintain the structure of muscle cells.

In the 20 mg/kg group, dystrophin levels reached 3.71% of normal, which is much higher than previous treatments. Patients also showed better movement and other important health improvements.

The treatment was mostly safe, with most side effects being mild or moderate. There were two cases of more serious side effects at a higher dose, but both patients recovered.

On September 23, Piper Sandler analyst Edward maintained an Outperform rating on Dyne (NASDAQ:DYN) with a $23 price target. The analyst recommended buying shares of the company as he expects new results from the Phase I/II ACHIEVE and DELIVER trials, along with the start of important registration studies by the end of 2024.

Tenthoff believes higher doses of DYNE-101 (5.4 mg/kg and 6.8 mg/kg) will lead to better results. Although Dyne’s (NASDAQ:DYN) stock dropped due to serious side effects from the 40 mg/kg dose of DYNE-251, the 20 mg/kg dose given every four weeks has shown important benefits, like effective exon skipping and improved dystrophin levels.