Investors have loaded Alethio Therapeutics, a U.K.-based biotechnology startup, with $90 million in funding to advance its treatments for a group of rare blood cancers, the company announced Thursday.

该公司周四宣布，英国生物技术初创企业Alethio Therapeutics已获得9,000万美元融资，用于推进其针对一组罕见血液癌症的治疗方案。

Alethio is diving headfirst into myeloproliferative neoplasms, a group of slow-progressing hematological malignancies that include primary myelofibrosis, essential thrombocythemia and polycythemia vera. In those conditions, the body creates too many blood cells, triggering a host of health problems such as clots, bone marrow scarring or heart attacks..

Alethio 正全力投入骨髓增殖性肿瘤的研究，这是一组进展缓慢的血液系统恶性肿瘤，包括原发性骨髓纤维化、原发性血小板增多症和真性红细胞增多症。在这些疾病中，身体会产生过多的血细胞，从而引发一系列健康问题，如血栓、骨髓纤维化（瘢痕形成）或心肌梗死。

“With these patients, it's a bit like an accelerator stuck down, constant drive, no brake,” said Rohit Batta, Alethio's CEO. “But the car doesn’t crash immediately. It goes for far too long.”

“对于这些患者来说，情况有点像油门卡住，持续加速，没有刹车，”Alethio首席执行官Rohit Batta表示，“但汽车并不会立即撞毁，而是会行驶过长的时间。”

Myeloproliferative neoplasms can be managed with frequent blood withdrawals, hydroxyurea, interferon therapies or “JAK” inhibitors such as Jakafi. And more recently, a new

骨髓增殖性肿瘤可通过频繁放血、羟基脲、干扰素疗法或“JAK”抑制剂（如Jakafi）进行管理。而最近，一种新的

Takeda medicine, Mimrylo

武田药品，Mimrylo

, was approved for polycythemia vera. But existing treatments still don’t help the majority of people with these conditions, Batta said.

，已获批用于治疗真性红细胞增多症。但巴塔表示，现有治疗方法仍然无法帮助大多数患有这些疾病的患者。

Rohit Batta is the CEO of Alethio Therapeutics.

Rohit Batta 是 Alethio Therapeutics 的首席执行官。

Permission granted by Alethio Therapeutics

Alethio Therapeutics 授权许可

Alethio’s lead program, dubbed “ATX-011,” is a monoclonal antibody Batta’s team theorizes can address those left behind, offering what the biotech says is an advantage over other entrants in a field that includes Jakafi’s maker

Alethio 的旗舰项目“ATX-011”是一种单克隆抗体，Batta 的团队认为它能够惠及那些被现有疗法遗漏的患者群体。这家生物技术公司称，这使其在包括 Jakafi 制造商在内的竞争领域中具备优势。

Incyte

Incyte

,

，

Merck & Co.

默克公司

and

和

Novartis

诺华

.

。

Rather than rely on what Batta calls a “blunt” approach, ATX-011 homes in on G6b, a receptor found on the surface of platelets and certain bone marrow cells. Importantly, G6b is only expressed on cells known to induce myeloproliferative neoplasms or cause bone marrow scarring, suggesting that a drug binding to the protein might have a precise impact on these diseases.

ATX-011 没有依赖巴塔所称的“粗放”方法，而是精准靶向 G6b，这是一种存在于血小板和某些骨髓细胞表面的受体。重要的是，G6b 仅在已知会诱发骨髓增殖性肿瘤或导致骨髓纤维化的细胞上表达，这表明与该蛋白结合的药物可能对这些疾病产生精准的治疗作用。

Alethio believes the drug could be beneficial regardless of which disease-driving mutation patients have, too..

Alethio 认为，无论患者携带哪种致病突变，该药物都可能有益。

Preclinical studies have shown ATX-011 is “a well-behaved medicine,” Batta said. It is expected to enter clinical testing in the first half of 2028.

巴塔表示，临床前研究表明，ATX-011 是一种“表现良好的药物”。预计该药将于 2028 年上半年进入临床试验阶段。

“The ultimate aim for ATX-011 is to allow patients to go and live their daily lives,” Batta said.

“ATX-011的最终目标是让患者能够回归并过他们的日常生活，”巴塔说。

A second drug prospect, an antibody-drug conjugate, is also in development for myelofibrosis.

另一种候选药物——抗体偶联药物——也正在开发中，用于治疗骨髓纤维化。

Alethio’s Series A funding was co-led by Sofinnova Partners and RA Capital Management, and also backed by Cancer Research Horizons, Oxford Science Enterprises and the University of Oxford. The startup spun out of research at the university, and was co-founded by Adam Mead and Beth Psaila, a pair of physicians and professors working there. .

Alethio 的 A 轮融资由 Sofinnova Partners 和 RA Capital Management 联合领投，并获得了 Cancer Research Horizons、Oxford Science Enterprises 以及牛津大学的支持。这家初创公司脱胎于该大学的研究项目，由在该校工作的两位医师兼教授 Adam Mead 和 Beth Psaila 联合创立。

Graziano Seghezzi, a managing partner at Sofinnova Partners, said Alethio’s decision to target essential thrombocythemia could give its drug greater “reach” compared to approved treatments for other myeloproliferative neoplasms. Essential thrombocythemia affects an estimated 150,000 patients in the U.S., according to Alethio, more than the combined amounts believed to have .

Sofinnova Partners 的管理合伙人 Graziano Seghezzi 表示，Alethio 决定将原发性血小板增多症作为靶点，与其针对其他骨髓增殖性肿瘤的已获批疗法相比，可能使其药物具有更广泛的“覆盖范围”。据 Alethio 称，原发性血小板增多症在美国影响的患者估计达 15 万人，超过被认为患有其他骨髓增殖性肿瘤的患者总数。

polycythemia vera

真性红细胞增多症

and

和

primary myelofibrosis

原发性骨髓纤维化

.

。

“The key words are we're not controlling the disease, we're treating it,” Seghezzi said.

“关键词在于，我们并非在控制这种疾病，而是在治疗它。”塞盖齐表示。