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SMALL CAP IDEA: Why 投資家s should 追加する Hemogenyx 製薬のs to their watchlist

By Ian Lyall At Proactive 投資家s For Thisismoney.co.uk

Updated: 14:05 BST, 15 April 2024

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In a 最近の article, I said at least 20 micro-cap 生命科学s 在庫／株s were waiting to pop once 利益／興味 returned to the 部門.

投資家s can now 追加する Hemogenyx 製薬のs?to the watch 名簿（に載せる）／表（にあげる） after it pulled off the 近づく impossible by raising $4.2million at a time when 投資家s have 大部分は gone into hibernation.

This feat of resourcefulness, which will 基金 the 初期の行う／開催する／段階s of a first-in-human 熟考する／考慮する of the 麻薬 developer's lead 資産, HEMO-CAR-T, only tells part of the story.

The pedigree of the 科学の team 支援 the 成果／努力s of Hemogenyx is impressive.

At the same time, the 利益／興味 in the next 世代 of CAR-T ? the type of therapy that Hemogenyx is developing ? amongst big pharma has seen the likes of AstraZeneca, Johnson & Johnson, Abbvie and Novartis 最高の，を越す the billion-dollar threshold in milestone and 王族支払い（額）s.

The pedigre e of the 科学の team 支援 the 成果／努力s of Hemogenyx is impressive

Upfront 支払い（額）s, usually a good litmus 実験（する） of 'real' 利益／興味 from these 巨人s of the world of 麻薬s, have been 注目する，もくろむ-watering; in some 事例／患者s up to $100million.

The 推論する／理由? CAR-T 申し込む／申し出s something that the healthcare 産業 rarely comes up with ? a curative 治療, showing the world has truly moved on from the 時代 of me-too, once-a-day pills such as statins.

Anyway, before we delve into the nuts and bolts of the Hemogenyx 投資事例／患者, it is probably 価値（がある） explaining just what CAR-T therapy is and how it might revolutionise the 治療 of 癌s such as 激烈な／緊急の myeloid leukaemia (AML), which is the particular area of 利益／興味 of this UK biotech.

CAR-T, or chimeric antigen receptor T-独房 therapy, redefines the 戦う／戦い against the 病気 by reprogramming the 団体／死体's own natural defences, called T-独房s, to recognise and kill 癌独房s.

This 前進するd form of immunotherapy 伴う／関わるs 抽出するing T-独房s from a 患者, genetically 工学 them to 的癌独房s, and then 再提出するing these 高めるd 独房s into the 患者's bloodstream.

CAR-T has 論証するd remarkable success in 取り組むing 確かな血癌s, 申し込む／申し出ing new avenues for 戦闘ing 以前 untreatable 癌s.

にもかかわらず its 可能性のある, much of the 研究 is now 焦点（を合わせる）d on CAR-T therapy's 重要な味方する影響s, while broadening its applicability to a wider array of 癌s.

Hemogenyx will use the newly raised 基金s to take HEMO-CAR-T into the clinic to 扱う／治療する AML later this year.

The open-label 熟考する／考慮する will 実験（する） for any 可能性のある味方する影響s from 扱う／治療するing people with 血-borne 病気s and 査定する／（税金などを）課す how the therapy interacts with the 団体／死体.

A 第２位 goal or 'endpoint' will be whether any of the 批判的に ill AML 患者s chosen for the 裁判，公判答える／応じる to the CAR-T infusion.

Under the strict 指導基準s laid 負かす／撃墜する by the US Food & 麻薬行政, 患者s will be dosed 個々に one month apart, recognising people are 存在治めるd with a 潜在的に有毒な独房s.

Hemogenyx hopes to have the results from 'two, or even three 患者s' in time for the American Society of Hematology 会議／協議会 in 早期に December.

Success at this 行う／開催する／段階 would be a 30% 返答率, says Dr Vladislav Sandler, 長，指導者 (n)役員／(a)執行力のある and co-創立者 of Hemogenyx.

Investigational work will be carried out by a team from the University of Pennsylvania, led by Professor Nolle Frey, who has 長，率いるd a number of important CAR-T 臨床の裁判，公判s.

The entre was 供給するd by Professor Carl June, the 'father' of CAR-T, and his 同僚, Dr Saar Gill, who was 伴う／関わるd in the preclinical 研究 on HEMO-CAR-T.

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