Bilateral Vestibulopathy

FRAMINGHAM, Mass.--(BUSINESS WIRE)-- Variantyx, a leader in genomic precision medicine, today announced that the set of short tandem repeat (STR) expansions detected by its Genomic Unity® line of whole genome-based tests has been expanded. With these additions, Variantyx becomes one of the first laboratories to offer testing for two recently characterized ataxia variants: the biallelic AAGGG expansion in RFC1 that causes cerebellar ataxia, neuropathy, vestibular areflexia syndrome (CANVAS) and the monoallelic, deep intronic GAA expansion in FGF14 that causes spinocerebellar ataxia 27B (SCA27B). “Short tandem repeat gene analysis and interpretation has always been challenging in clinical diagnostics,” said Christine Stanley, PhD, FACMG, Chief Director of Clinical Genomics at Variantyx. “Because the entire genome is sequenced, Genomic Unity® testing has the ability to detect a broader range of STRs from a single sample compared to targeted technologies utilized by other laboratories. At Variantyx we’re committed to continually extending the detection capabilities of our whole genome-based platform and are pleased to now be able to provide extended analysis and interpretation of STRs which is expected to result in additional diagnostic answers for patients.” The product line now analyzes an industry-leading 37 repeat expansions, collectively enabling the genetic diagnosis of late-onset neuropathies, neuromuscular disorders and movement disorders - particularly ataxias - as well as early-onset disorders, including those with symptoms of epilepsy and intellectual disability. The additional detection capabilities are automatically included in all Genomic Unity® comprehensive analyses and relevant targeted analyses. About Variantyx Variantyx is an award-winning, technology-driven precision medicine company providing disruptive solutions for the genetic disorders, reproductive health, and precision oncology markets. The proprietary whole genome analysis platforms developed by Variantyx allow clinicians and patients to better understand a person’s genetic makeup, leading to unmatched diagnostic capabilities and improved personalized treatment recommendations. For more information, please visit . View source version on businesswire.com: Contacts Haim Neerman Haim.neerman@variantyx.com (617) 209-2090 Source: Variantyx View this news release online at:

While Regeneron has been working with Decibel Therapeutics for some time, the deal underscores a trend as major pharmaceutical companies look to hearing loss biotechs to pad their pipelines. Regeneron, listening to the call for more gene therapies, has added hearing loss specialist Decibel Therapeutics to its playlist in a deal valuing the biotech at $109 million at closing. Regeneron and Decibel were already working together through a 2017 collaboration for three gene therapies for different forms of congenital, monogenic hearing loss. Decibel’s lead hearing loss candidate is DB-OTO, which is currently moving through a phase 1/2 trial called CHORD for individuals with hearing loss caused by mutations of the otoferlin gene. Other assets include preclinical programs AAV.103 for GJB2-related hearing loss and AAV.104 for people with stereocilin-related hearing loss. The biotech also specializes in balance. The deal has a total equity value of $213 million including contingent value rights (CVRs) if certain enrollment, dosing and regulatory milestones for DB-OTO are met. Decibel will come under Regeneron’s wing for $4 per share with the additional non-tradeable CVRs for $3.50 apiece. One of those milestones is the acceptance of a regulatory application in a number of countries by the end of 2028. Regeneron, most well known for its antibody work, sees big opportunity in gene therapy and hearing loss specifically. The company noted that while otoferlin hearing loss itself is rare, most congenital hearing loss diagnosed in developed countries results from a single gene defect. That means these patients could be targeted with gene therapy. George Yancopoulos, M.D., Ph.D., Regeneron’s chief scientific officer and president, said DB-OTO will soon be administered to patients in the early-stage trial and could provide the proof-of-concept needed to advance future gene therapies for more common types of hearing loss. In addition to the gene therapy portfolio, Decibel also has DB-020 in its pipeline, a formulation of sodium thiosulfate being tested in a phase 1b for patients with hearing loss caused by the chemotherapy cisplatin. In June 2022, Decibel presented data showing that the therapy protected patients from hearing loss in the treated ear. The company is also developing two hair cell regeneration programs for the balance disorder bilateral vestibulopathy and for sensorineural hearing loss. While Regeneron has been working with Decibel for some time, the deal underscores the accelerating interest in hearing loss biotechs. In October 2022, Eli Lilly picked up Decibel’s lead rival, Akouos, for $487 million upfront. That company is also using gene therapy to target otoferlin hearing loss. And just a month before that, Fennec Pharmaceuticals got a chemotherapy-induced hearing loss treatment approved by the FDA after decades in development.  Decibel launched its hearing loss mission in 2015 and is led by Laurence Reid, Ph.D., who joined the company in 2020. “After full consideration, the Decibel board has determined that this transaction is the best way to maximize shareholder value and ultimately benefit patients,” Reid said in a statement. “We have collaborated with our colleagues at Regeneron for the past six years and have huge respect for their research and development capabilities. We have full confidence that with Regeneron’s expertise and resources the Decibel pipeline can be optimally developed, and our team is committed to enabling that long-term success.” Decibel had cash and equivalents of $87.9 million as of March 31, which was expected to last into the first half of 2024. The biotech's shares were up 71% to $4.78 compared to a prior close of $2.79 in premarket trading Wednesday, suggesting investors like the tune of the Regeneron deal. 

Platform technology company Replay has launched Eudora, a gene therapy company targeting genetic diseases of the retina. Eudora will utilize Replay’s high payload capacity herpes simplex virus (HSV) vector to deliver gene therapy payloads, the company announced Monday. Replay plans to launch four companies using this technology, although Eudora is the first to launch. The other three will focus on brain, skin and muscle diseases. Eudora’s targets include retinitis pigmentosa, Stargardt disease and Usher syndrome type 1B (USH1). Retinitis pigmentosa is a family of rare eye diseases that cause the cells in the retina to break down, resulting in vision loss. Stargardt disease is also a rare genetic disorder caused by the buildup of fatty material on the macula, the part of the retina required for sharp, central vision. USH1 is marked by congenital, bilateral, profound sensorineural hearing loss, vestibular areflexia, and adolescent-onset retinitis pigmentosa. “Replay’s differentiated corporate structure allows its platform technologies to be applied to indications in areas of high unmet medical need across a portfolio of focused product companies,” stated Adrian Woolfson, executive chairman, president and co-founder of Replay. Eudora’s four co-founders include Joe Glorioso, Ph.D., Mark Blumenkranz, M.D., David Schaffer, Ph.D. and Vinit Mahajan, M.D., Ph.D. Glorioso is the synHSV senior advisor at Replay. He is a professor in the microbiology and molecular genetics department at the University of Pittsburgh School of Medicine. Glorioso stated that Eudora's HSV-1 delivery platform was developed by his team at the University of Pittsburgh over several decades. The most common gene therapy vector is the adeno-associated virus (AAV). Replay and Eudora indicate the advantage of the synHSV is its ability to deliver payloads eight times the size of AAV vector payloads. Replay is also working on an HSV vector that it believes can provide a payload 30 times larger than AAV vectors can handle. Replay calls itself a genome writing company. It has a hub-and-spoke model, identifying specific technology platforms and launching companies to utilize them. The company launched in July with $55 million in seed financing. The round was led by KKR and OMX Ventures, with participation from ARTIS Ventures and Lansdowne Partners, SALT, DeciBio Ventures, and Axial.