Biotechs developing therapeutics to treat rare diseases include two that received FDA approvals this year for drugs to treat spinal muscular atrophy and Alexander disease.
Biopharma professionals who want to work in rare diseases—which can be life altering or life threatening for relatively small patient populations—may want to consider companies that have or are nearing FDA approvals.
Here are four biotechs making strides toward bringing much-needed drugs to market that are hiring.
Scholar Rock
Based in Cambridge, Massachusetts, Scholar Rock is developing therapies for people living with serious diseases, including spinal muscular atrophy (SMA) and facioscapulohumeral muscular dystrophy (FSHD). Founded in 2012, the biotech, which currently has 30 open positions, uses a proprietary platform to selectively target protein growth factors before they are activated in the body.
Last month, lead asset apitegromab-mtsn, now called Isembyld, received FDA approval for the treatment of SMA in adults and children who are at least 2 years old and are also undergoing SMN2-targeted treatment. Scholar Rock’s monoclonal antibody is the first muscle-targeted therapy that improves motor function in patients who have SMA, a rare genetic illness characterized by the progressive weakening of muscles.
The biotech has also been granted FDA fast track and orphan drug designations for apitegromab for the treatment of FSHD, a rare hereditary neuromuscular disease characterized by muscle atrophy, weakness and functional decline. Participant dosing is underway in the Phase 2 FORGE clinical trial evaluating the candidate.
In addition to rare diseases, Scholar Rock is also working to advance preclinical and clinical programs for locally advanced or metastatic solid tumors, multiple fibrotic indications and iron-restricted anemia.
The company’s 30 open positions include senior manager/associate director, biostatistics; associate director/director, clinical operations compliance; and director, global quality control.
Insmed
Founded in 1988, Insmed is developing therapies in three therapeutic areas: respiratory, immunology and inflammation, and neuro and other rare diseases. In that final category, disease areas include ALS and Duchenne muscular dystrophy (DMD). The Bridgewater, New Jersey–based company, which lists over 100 positions open, has two approved therapies, including antibacterial drug Arikayce, used for nontuberculous mycobacterial (NTM) lung disease, a rare disorder most common in people with long-term lung conditions.
Within the neuro and other rare diseases space, Insmed’s most advanced candidates are gene therapies INS1201 and INS1202 for the treatment of DMD and ALS, respectively. DMD is a neuromuscular disorder in which muscle cells weaken, while ALS is a degenerative neurological disorder that affects nerve cells in the brain and spinal cord. Insmed is enrolling patients in the Phase 1 ASCEND clinical study of INS1201 and Phase 1 ARMOR study of INS1202.
The company counts among its areas of innovation a proprietary deimmunization platform that uses AI to reengineer proteins with the goal of silencing immunogenic properties while maintaining therapeutic activity. Insmed is using the platform to explore the feasibility of developing redosable gene therapies that could unlock new, high unmet need disease targets and potentially less immunogenic protein therapeutics.
The biotech’s over 100 open positions include associate director, clinical trial operations; senior scientist, analytical development-small molecule; and senior director, regulatory affairs CMC.
Ionis Pharmaceuticals
Founded in 1989, Carlsbad, California–based Ionis Pharmaceuticals, which has 35 open positions, is developing RNA- and DNA-targeted therapies for the treatment of serious diseases across its focus areas of neurology and cardiology. Rare and ultrarare diseases addressed by its clinical programs include Angelman syndrome and Alexander disease.
Ionis received FDA approval last month for antisense oligonucleotide (ASO) therapy Zanvastro, formerly zilganersen, the biotech’s first independent neurology drug. Zanvastro is the first therapy that directly targets the protein buildup that drives Alexander disease, an extremely rare leukodystrophy affecting the central nervous system.
Also in September, Ionis and partner Otsuka Pharmaceuticals reported positive data from the Phase 3 FUSION trial of another ASO, ulefnersen, in patients with a rare, genetic subtype of ALS. The therapy showed a statistically significant improvement versus placebo in functional impairment and survival in 73 patients. Ionis and Otsuka are exploring potential pathways for expedited approval of ulefnersen.
In another win this year, Ionis received FDA approval for antisense oligonucleotide Tryngolza to help control blood lipid levels and pancreatitis risk in patients with severe hypertriglyceridemia, a condition in which triglycerides in the blood are dangerously high. Tryngolza was already approved to help reduce triglycerides in adults with familial chylomicronemia syndrome, a rare form of severe hypertriglyceridemia.
Ionis’ 35 open positions include protein scientist, assistant director or associate director; manager/senior manager, GMP quality-drug substance; and assistant/associate director, pharmacovigilance quality assurance.
Dyne Therapeutics
Based in Waltham, Massachusetts, and founded in 2018, Dyne Therapeutics is developing therapies for the treatment of genetically driven neuromuscular diseases. The company, which has around 60 open positions, is advancing clinical programs for DMD and myotonic dystrophy type 1 (DM1) as well as preclinical programs for FSHD, Pompe disease and multiple DMD mutations. In its work, Dyne uses a proprietary platform consisting of an antigen-binding fragment (Fab), a linker and a payload that is rationally designed to target the genetic basis of diseases.
In July, the FDA accepted the biotech’s biologics license application seeking accelerated approval of investigational therapeutic zeleciment rostudirsen, or z-rostudirsen, to treat DMD amenable to exon 51 skipping. The agency set a target action date of Jan. 21, 2027. The FDA is not planning an advisory committee meeting for z-rostudirsen, Dyne said at the time.
Also in July, the agency approved Dyne’s investigational new drug application for DYNE-302 in FSHD, allowing the biotech to move forward with a Phase 1 clinical trial in adults with the disease who are able to walk. The company plans to pursue a traditional approval pathway in the U.S. for the therapeutic.
Dyne’s approximately 60 open positions include associate director, oligonucleotide development; scientist, RNA chemistry; and executive director, regulatory affairs.
